Breakthrough Therapies

The Breakthrough therapies chart is a list of all publicly announced breakthrough therapy designations since the program’s inception in 2012. The chart is updated daily and includes information about each individual agent or trade name of a drug or treatment, the sponsor company, the date of the designation’s public disclosure, the individual agent’s indication, or the broad disease category in which the designation was granted. The interactive search and sort feature lets you filter your results and download the table at any time into an excel format. Click here to learn more about the history of Breakthrough therapies. As of December 15, 2020 the FDA has approved 190* breakthrough therapy designated products and lists that there have been 1041 total requests for the designation with 399** designations granted.

Category
Date of BT Designation Disclosure
Approval Date
FDA Status
Trade Name (Agent) Sponsor Date of BT Designation Disclosure Approval Date Indication Category
 Ligelizumab (QGE031) Novartis International AG 1/14/2021 Treatment of chronic spontaneous urticaria (CSU), also known as chronic idiopathic urticaria (CIU), in patients who have an inadequate response to H1-antihistamine treatment. Rare Inherited Disorders
Date of BT Designation Disclosure: 1/14/2021/2021
Approval Date:
Indication: Treatment of chronic spontaneous urticaria (CSU), also known as chronic idiopathic urticaria (CIU), in patients who have an inadequate response to H1-antihistamine treatment.
Category: Rare Inherited Disorders
 APL-106 (uproleselan) Apollomics, Inc 1/07/2021 Treatment of relapsed/refractory acute myeloid leukemia (AML). Cancer
Date of BT Designation Disclosure: 1/07/2021/2021
Approval Date:
Indication: Treatment of relapsed/refractory acute myeloid leukemia (AML).
Category: Cancer
 Chiauranib Shenzhen Chipscreen Biosciences Co., Ltd 1/05/2021 "Treatment of patients with small cell lung cancer (SCLC) with disease progression or recurrence after two lines of prior systematic chemotherapy. " Cancer
Date of BT Designation Disclosure: 1/05/2021/2021
Approval Date:
Indication: "Treatment of patients with small cell lung cancer (SCLC) with disease progression or recurrence after two lines of prior systematic chemotherapy. "
Category: Cancer
  Tiragolumab in combination with Tecentriq The Roche Group 1/05/2021 First-line treatment of people with metastatic non-small cell lung cancer (NSCLC) whose tumours have high PD-L1 expression with no EGFR or ALK genomic tumour aberrations Cancer
Date of BT Designation Disclosure: 1/05/2021/2021
Approval Date:
Indication: First-line treatment of people with metastatic non-small cell lung cancer (NSCLC) whose tumours have high PD-L1 expression with no EGFR or ALK genomic tumour aberrations
Category: Cancer
  CNCT19 (CD19 CAR-T) CASI Pharmaceuticals, Inc.  12/23/2020 A potential CD19 CAR-T therapy for the treatment of adults with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL) Cancer
Date of BT Designation Disclosure: 12/23/2020/2020
Approval Date:
Indication: A potential CD19 CAR-T therapy for the treatment of adults with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL)
Category: Cancer
 IO102 and IO103 with anti-PD-1 mAb IO Biotech 12/15/2020 Patients with unresectable/ metastatic melanoma Cancer
Date of BT Designation Disclosure: 12/15/2020/2020
Approval Date:
Indication: Patients with unresectable/ metastatic melanoma
Category: Cancer
 IO102 and IO103 with anti-PD-1 mAb IO Biotech 12/15/2020 Patients with unresectable/ metastatic melanoma Cancer
Date of BT Designation Disclosure: 12/15/2020/2020
Approval Date:
Indication: Patients with unresectable/ metastatic melanoma
Category: Cancer
 CUTX-101 (Copper Histidinate) Cyprium Therapeutics, Inc. 12/15/2020 Treatment of Menkes Disease Rare Inherited Disorders
Date of BT Designation Disclosure: 12/15/2020/2020
Approval Date:
Indication: Treatment of Menkes Disease
Category: Rare Inherited Disorders
 Pegloprastide (AVB-620) Avelas Biosciences 12/14/2020 Intraoperative detection and visualization of positive margins during breast cancer surgery. Cancer
Date of BT Designation Disclosure: 12/14/2020/2020
Approval Date:
Indication: Intraoperative detection and visualization of positive margins during breast cancer surgery.
Category: Cancer
 Sotorasib Amgen 12/08/2020 Treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with KRAS G12C mutation, as determined by an FDA-approved test, following at least one prior systemic therapy. Cancer
Date of BT Designation Disclosure: 12/08/2020/2020
Approval Date:
Indication: Treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with KRAS G12C mutation, as determined by an FDA-approved test, following at least one prior systemic therapy.
Category: Cancer
 Repotrectinib Turning Point Therapeutics Inc. 12/08/2020 "Treatment of patients with ROS1-positive metastatic non-small cell lung cancer (NSCLC) who have not been treated with a ROS1 tyrosine kinase inhibitor (TKI-naïve). " Cancer
Date of BT Designation Disclosure: 12/08/2020/2020
Approval Date:
Indication: "Treatment of patients with ROS1-positive metastatic non-small cell lung cancer (NSCLC) who have not been treated with a ROS1 tyrosine kinase inhibitor (TKI-naïve). "
Category: Cancer
 Zanidatamab Zymeworks Inc. 11/30/2020 Treatment for patients with previously-treated HER2 gene-amplified biliary tract cancer (BTC). Cancer
Date of BT Designation Disclosure: 11/30/2020/2020
Approval Date:
Indication: Treatment for patients with previously-treated HER2 gene-amplified biliary tract cancer (BTC).
Category: Cancer
 Zanidatamab Zymeworks Inc. 11/30/2020 Treatment for patients with previously-treated HER2 gene-amplified biliary tract cancer (BTC). Cancer
Date of BT Designation Disclosure: 11/30/2020/2020
Approval Date:
Indication: Treatment for patients with previously-treated HER2 gene-amplified biliary tract cancer (BTC).
Category: Cancer
 Vixarelimab Kiniksa Pharmaceuticals Ltd 11/16/2020 Treatment of pruritus associated with prurigo nodularis, a chronic inflammatory skin condition characterized by severely pruritic skin nodules Other
Date of BT Designation Disclosure: 11/16/2020/2020
Approval Date:
Indication: Treatment of pruritus associated with prurigo nodularis, a chronic inflammatory skin condition characterized by severely pruritic skin nodules
Category: Other
 Olorofim  F2G Ltd 10/22/2020 Treatment of Central Nervous System (CNS) coccidioidomycosis refractory or otherwise unable to be treated with standard of care therapy Infectious Disease
Date of BT Designation Disclosure: 10/22/2020/2020
Approval Date:
Indication: Treatment of Central Nervous System (CNS) coccidioidomycosis refractory or otherwise unable to be treated with standard of care therapy
Category: Infectious Disease
 Lanifibranor Inventiva 10/12/2020 Treatment of non-alcoholic steatohepatitis (NASH), mucopolysaccharidoses (MPS) Other
Date of BT Designation Disclosure: 10/12/2020/2020
Approval Date:
Indication: Treatment of non-alcoholic steatohepatitis (NASH), mucopolysaccharidoses (MPS)
Category: Other
 IMGN632 ImmunoGen, Inc 10/05/2020 Treatment of relapsed or refractory blastic plasmacytoid dendritic cell neoplasm Cancer
Date of BT Designation Disclosure: 10/05/2020/2020
Approval Date:
Indication: Treatment of relapsed or refractory blastic plasmacytoid dendritic cell neoplasm
Category: Cancer
 Farxiga® (dapagliflozin) AstraZeneca  10/02/2020 Patients with chronic kidney disease (CKD), with and without type 2 diabetes (T2D) Other
Date of BT Designation Disclosure: 10/02/2020/2020
Approval Date:
Indication: Patients with chronic kidney disease (CKD), with and without type 2 diabetes (T2D)
Category: Other
 AXS-12 Axsome Therapeutics 8/05/2020 Treatment of cataplexy in patients with narcolepsy. Other
Date of BT Designation Disclosure: 8/05/2020/2020
Approval Date:
Indication: Treatment of cataplexy in patients with narcolepsy.
Category: Other
 Tagrisso (osimertinib) AstraZeneca 7/30/2020 12/18/2020 Adjuvant treatment of patients with early-stage (IB, II and IIIA) epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC) after complete tumor resection with curative intent. Cancer
Date of BT Designation Disclosure: 7/30/2020/2020
Approval Date: 2020
Indication: Adjuvant treatment of patients with early-stage (IB, II and IIIA) epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC) after complete tumor resection with curative intent.
Category: Cancer
 Pevonedistat Takeda Pharmaceutical Company 7/30/2020 Treatment of patients with higher-risk myelodysplastic syndromes (HR-MDS) Cancer
Date of BT Designation Disclosure: 7/30/2020/2020
Approval Date:
Indication: Treatment of patients with higher-risk myelodysplastic syndromes (HR-MDS)
Category: Cancer
 MK-6482 Merck Sharpe & Dohme Corp. 7/29/2020 Treatment of patients with von Hippel-Lindau (VHL) disease-associated renal cell carcinoma (RCC) with nonmetastatic RCC tumors less than three centimeters in size, unless immediate surgery is required. Cancer
Date of BT Designation Disclosure: 7/29/2020/2020
Approval Date:
Indication: Treatment of patients with von Hippel-Lindau (VHL) disease-associated renal cell carcinoma (RCC) with nonmetastatic RCC tumors less than three centimeters in size, unless immediate surgery is required.
Category: Cancer
 Mavacamten (MYK-461) MyoKardia 7/23/2020 Treatment of symptomatic, obstructive hypertrophic cardiomyopathy (HCM). Rare Inherited Disorders
Date of BT Designation Disclosure: 7/23/2020/2020
Approval Date:
Indication: Treatment of symptomatic, obstructive hypertrophic cardiomyopathy (HCM).
Category: Rare Inherited Disorders
 CD20xCD3 F. Hoffmann-La Roche AG 7/14/2020 Treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma who have received at least two prior systemic therapies Cancer
Date of BT Designation Disclosure: 7/14/2020/2020
Approval Date:
Indication: Treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma who have received at least two prior systemic therapies
Category: Cancer
 CTP-543 Concert Pharmaceuticals 7/08/2020 Treatment of Alopecia Areata Rare Inherited Disorders
Date of BT Designation Disclosure: 7/08/2020/2020
Approval Date:
Indication: Treatment of Alopecia Areata
Category: Rare Inherited Disorders
 ER-004 Esperare 7/06/2020 Investigational Prenatal Treatment ER-004 In X-Linked Hypohidrotic Ectodermal Dysplasia Rare Inherited Disorders
Date of BT Designation Disclosure: 7/06/2020/2020
Approval Date:
Indication: Investigational Prenatal Treatment ER-004 In X-Linked Hypohidrotic Ectodermal Dysplasia
Category: Rare Inherited Disorders
 TLX250-CDX Telix Pharmaceuticals 7/01/2020 Renal cancer imaging product Cancer
Date of BT Designation Disclosure: 7/01/2020/2020
Approval Date:
Indication: Renal cancer imaging product
Category: Cancer
 AXS-05 Axsome Therapeutics 6/26/2020 Treatment of Alzheimer’s disease agitation, major depressive disorder, and other central nervous system (CNS) disorders Other
Date of BT Designation Disclosure: 6/26/2020/2020
Approval Date:
Indication: Treatment of Alzheimer’s disease agitation, major depressive disorder, and other central nervous system (CNS) disorders
Category: Other
 Brensocatib Insmed Incorporated 6/18/2020 Treatment for bronchiectasis Rare Inherited Disorders
Date of BT Designation Disclosure: 6/18/2020/2020
Approval Date:
Indication: Treatment for bronchiectasis
Category: Rare Inherited Disorders
 Brensocatib (INS1007) Insmed Incorporated 6/08/2020 Treatment of bronchiectasis and other inflammatory diseases and of adult patients with non-cystic fibrosis bronchiectasis (NCFBE) for reducing exacerbation Rare Inherited Disorders
Date of BT Designation Disclosure: 6/08/2020/2020
Approval Date:
Indication: Treatment of bronchiectasis and other inflammatory diseases and of adult patients with non-cystic fibrosis bronchiectasis (NCFBE) for reducing exacerbation
Category: Rare Inherited Disorders
 HSV Pritelivir (AIC316) Anti-infective Cures GmbH 6/05/2020 Treatment of acyclovir-resistant mucocutaneous herpes simplex virus (HSV) infections in immunocompromised adults Other
Date of BT Designation Disclosure: 6/05/2020/2020
Approval Date:
Indication: Treatment of acyclovir-resistant mucocutaneous herpes simplex virus (HSV) infections in immunocompromised adults
Category: Other
 Neuromodulation therapy (NTX100) Noctrix Health 6/02/2020 Treatment of adults with primary moderate-severe Restless Legs Syndrome (RLS) who are refractory to medications Other
Date of BT Designation Disclosure: 6/02/2020/2020
Approval Date:
Indication: Treatment of adults with primary moderate-severe Restless Legs Syndrome (RLS) who are refractory to medications
Category: Other
 Enhertu (trastuzumab deruxtecan) AstraZeneca plc and Daiichi Sankyo Co. 5/11/2020 1/18/2021 Treatment of adult patients with previously treated HER2 positive advanced gastric cancer Cancer
Date of BT Designation Disclosure: 5/11/2020/2020
Approval Date: 2021
Indication: Treatment of adult patients with previously treated HER2 positive advanced gastric cancer
Category: Cancer
 Bavencio (avelumab) Merck Sharpe & Dohme Corp. 4/09/2020 7/01/2020 Patients with locally advanced or metastatic urothelial carcinoma that has not progressed with first-line platinum-containing chemotherapy Cancer
Date of BT Designation Disclosure: 4/09/2020/2020
Approval Date: 2020
Indication: Patients with locally advanced or metastatic urothelial carcinoma that has not progressed with first-line platinum-containing chemotherapy
Category: Cancer
 Sotatercept Acceleron Pharma 4/08/2020 Patients with pulmonary arterial hypertension Cardiovascular
Date of BT Designation Disclosure: 4/08/2020/2020
Approval Date:
Indication: Patients with pulmonary arterial hypertension
Category: Cardiovascular
 Baricitinib Eli Lilly and Company + Incyte Corporation 3/16/2020 Patients with alopecia areata Other
Date of BT Designation Disclosure: 3/16/2020/2020
Approval Date:
Indication: Patients with alopecia areata
Category: Other
 JNJ-6372 Janssen Biotech, Inc. 3/10/2020 Patients with EGFR-positive metastatic non-small cell lung cancer who harbor exon 20 insertion mutations, and whose disease has progressed on or after platinum-based chemotherapy Cancer
Date of BT Designation Disclosure: 3/10/2020/2020
Approval Date:
Indication: Patients with EGFR-positive metastatic non-small cell lung cancer who harbor exon 20 insertion mutations, and whose disease has progressed on or after platinum-based chemotherapy
Category: Cancer
  Yervoy (ipilimumab) Bristol-Myers Squibb Company 3/10/2020 In combination with nivolumab, for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib Cancer
Date of BT Designation Disclosure: 3/10/2020/2020
Approval Date:
Indication: In combination with nivolumab, for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib
Category: Cancer
 Esbriet (pirfenidone) Hoffman-La Roche Inc. 3/03/2020 Patients with unclassifiable interstitial lung disease Other
Date of BT Designation Disclosure: 3/03/2020/2020
Approval Date:
Indication: Patients with unclassifiable interstitial lung disease
Category: Other
 Exebacase ContraFect Corporation 2/24/2020 Patients with MRSA bacteremia, including right-sided endocarditis, when used in conjunction with standard of care antibiotics Infectious Disease
Date of BT Designation Disclosure: 2/24/2020/2020
Approval Date:
Indication: Patients with MRSA bacteremia, including right-sided endocarditis, when used in conjunction with standard of care antibiotics
Category: Infectious Disease
 Padcev Astellas Pharma 2/20/2020 In combination with Keytruda for the treatment of patients with unresectable locally advanced or metastatic urothelial cancer who are unable to receive cisplatin-based chemotherapy in the first-line setting Cancer
Date of BT Designation Disclosure: 2/20/2020/2020
Approval Date:
Indication: In combination with Keytruda for the treatment of patients with unresectable locally advanced or metastatic urothelial cancer who are unable to receive cisplatin-based chemotherapy in the first-line setting
Category: Cancer
 APR-246 Aprea Therapeutics 1/30/2020 Treatment of patients with myelodysplastic syndromes with a susceptible TP53 mutation Cancer
Date of BT Designation Disclosure: 1/30/2020/2020
Approval Date:
Indication: Treatment of patients with myelodysplastic syndromes with a susceptible TP53 mutation
Category: Cancer
 Artesunate AMIVAS LLC 2020 5/26/2020 Initial treatment of severe malaria in adult and pediatric patients Other
Date of BT Designation Disclosure: 2020/2020
Approval Date: 2020
Indication: Initial treatment of severe malaria in adult and pediatric patients
Category: Other
 TEPEZZA (TEPROTUMUMABTRBW) HORIZON THERAPEUTICS IRELAND DAC 2020 1/21/2020 Treatment of thyroid eye disease Other
Date of BT Designation Disclosure: 2020/2020
Approval Date: 2020
Indication: Treatment of thyroid eye disease
Category: Other
 Qinlock (Ripretinib) DECIPHERA PHARMACEUTICALS LLC 2020 5/15/2020 Treatment of adult patients with advanced gastrointestinal stromal tumor (GIST) who have received prior treatment with 3 or more kinase inhibitors, including imatinib Cancer
Date of BT Designation Disclosure: 2020/2020
Approval Date: 2020
Indication: Treatment of adult patients with advanced gastrointestinal stromal tumor (GIST) who have received prior treatment with 3 or more kinase inhibitors, including imatinib
Category: Cancer
 Pemazyre (pemigatinib) Incyte Corporation 2020 2020 Treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test Cancer
Date of BT Designation Disclosure: 2020/2020
Approval Date: 2020
Indication: Treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test
Category: Cancer
 Opdivo (nivolumab) Bristol-Myers Squibb Company 2020 3/10/2020 In combination with ipilimumab, for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib Cancer
Date of BT Designation Disclosure: 2020/2020
Approval Date: 2020
Indication: In combination with ipilimumab, for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib
Category: Cancer
 B38M (JNJ‐4528) Legend Biotech + Janssen Global Services 12/06/2019 Patients with previously treated multiple myeloma Cancer
Date of BT Designation Disclosure: 12/06/2019/2019
Approval Date:
Indication: Patients with previously treated multiple myeloma
Category: Cancer
 Rilonacept Kiniksa 11/20/2019 Treatment of recurrent pericarditis Cardiovascular
Date of BT Designation Disclosure: 11/20/2019/2019
Approval Date:
Indication: Treatment of recurrent pericarditis
Category: Cardiovascular
 Inmazeb (atoltivimab, maftivimab, and odesivimab-ebgn) Regeneron Pharmaceuticals 11/19/2019 10/14/2020 Treatment for Zaire ebolavirus (Ebola virus) infection in adult and pediatric patients Infectious Disease
Date of BT Designation Disclosure: 11/19/2019/2019
Approval Date: 2020
Indication: Treatment for Zaire ebolavirus (Ebola virus) infection in adult and pediatric patients
Category: Infectious Disease
 Olorofim (F901318) F2G The Rare Fungal Disease Company 11/13/2019 Treatment of invasive mold infections in patients w limited or no treatment options, including aspergillosis refractory or intolerant to currently available therapy, & infections due to Lomentospora prolificans, Scedosporium, and Scopulariopsis species Other
Date of BT Designation Disclosure: 11/13/2019/2019
Approval Date:
Indication: Treatment of invasive mold infections in patients w limited or no treatment options, including aspergillosis refractory or intolerant to currently available therapy, & infections due to Lomentospora prolificans, Scedosporium, and Scopulariopsis species
Category: Other
 Ofev (nintedanib) Boehringer Ingelheim Pharmaceuticals 10/10/2019 3/09/2020 Chronic fibrosing interstitial lung diseases (ILDs) with a progressive phenotype Rare Inherited Disorders
Date of BT Designation Disclosure: 10/10/2019/2019
Approval Date: 2020
Indication: Chronic fibrosing interstitial lung diseases (ILDs) with a progressive phenotype
Category: Rare Inherited Disorders
 Zejula (niraparib) Janssen Pharmaceutica 10/03/2019 Patients with BRCA1/2-mutated metastatic castration-resistant prostate cancer (CRPC) who have previously received taxane chemotherapy and androgen receptor (AR)–targeted therapy Cancer
Date of BT Designation Disclosure: 10/03/2019/2019
Approval Date:
Indication: Patients with BRCA1/2-mutated metastatic castration-resistant prostate cancer (CRPC) who have previously received taxane chemotherapy and androgen receptor (AR)–targeted therapy
Category: Cancer
 Cotellic (cobimetinib) Memorial Sloan Kettering Cancer Center 10/03/2019 Adult patients with histiocytic neoplasms who do not harbor a BRAF V600 mutation Rare Inherited Disorders
Date of BT Designation Disclosure: 10/03/2019/2019
Approval Date:
Indication: Adult patients with histiocytic neoplasms who do not harbor a BRAF V600 mutation
Category: Rare Inherited Disorders
 tepotinib Merck 9/11/2019 Patients with metastatic non-small cell lung cancer (NSCLC) harboring a MET exon 14 skipping alteration who have progressed following platinum-based chemotherapy Cancer
Date of BT Designation Disclosure: 9/11/2019/2019
Approval Date:
Indication: Patients with metastatic non-small cell lung cancer (NSCLC) harboring a MET exon 14 skipping alteration who have progressed following platinum-based chemotherapy
Category: Cancer
 EBANGA ANSUVIMAB-ZYKL Ridgeback Biotherapeutics LP 9/06/2019 12/21/2020 Treatment for Ebola Infectious Disease
Date of BT Designation Disclosure: 9/06/2019/2019
Approval Date: 2020
Indication: Treatment for Ebola
Category: Infectious Disease
 TABRECTA (capmatinib) Novartis Pharmaceuticals Corporation 9/06/2019 5/06/2020 Treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have a mutation that leads to mesenchymal-epithelial transition (MET) exon 14 skipping as detected by an FDA-approved test Cancer
Date of BT Designation Disclosure: 9/06/2019/2019
Approval Date: 2020
Indication: Treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have a mutation that leads to mesenchymal-epithelial transition (MET) exon 14 skipping as detected by an FDA-approved test
Category: Cancer
 Nirogacestat SpringWorks Therapeutics, Inc. 8/29/2019 Adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis Cancer
Date of BT Designation Disclosure: 8/29/2019/2019
Approval Date:
Indication: Adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis
Category: Cancer
 NKTR-214 (bempegaldesleukin) with Opdivo (nivolumab) Nektar Therapeutics 8/23/2019 Untreated melanoma patients with either metastatic disease or who are unable to undergo surgery Cancer
Date of BT Designation Disclosure: 8/23/2019/2019
Approval Date:
Indication: Untreated melanoma patients with either metastatic disease or who are unable to undergo surgery
Category: Cancer
 Peginterferon Lambda Eiger Pharmaceuticals 8/20/2019 Hepatitis delta virus (HDV) infection Infectious Disease
Date of BT Designation Disclosure: 8/20/2019/2019
Approval Date:
Indication: Hepatitis delta virus (HDV) infection
Category: Infectious Disease
 Calquence (acalabrutinib) AstraZeneca UK Ltd 8/14/2019 11/21/2019 Adult patients with untreated chronic lymphocytic leukemia (CLL) Cancer
Date of BT Designation Disclosure: 8/14/2019/2019
Approval Date: 2019
Indication: Adult patients with untreated chronic lymphocytic leukemia (CLL)
Category: Cancer
 Calquence (acalabrutinib) AstraZeneca UK Ltd 8/14/2019 11/21/2019 Adult patients with relapsed or refractory chronic lymphocytic leukemia Cancer
Date of BT Designation Disclosure: 8/14/2019/2019
Approval Date: 2019
Indication: Adult patients with relapsed or refractory chronic lymphocytic leukemia
Category: Cancer
 trilaciclib 8/07/2019 Patients with small cell lung cancer Cancer
Date of BT Designation Disclosure: 8/07/2019/2019
Approval Date:
Indication: Patients with small cell lung cancer
Category: Cancer
 DCR-PHXC Dicerna Pharmaceuticals 7/15/2019 Primary hyperoxaluria type 1 (caused by a mutation in the AGXT gene) Other
Date of BT Designation Disclosure: 7/15/2019/2019
Approval Date:
Indication: Primary hyperoxaluria type 1 (caused by a mutation in the AGXT gene)
Category: Other
 Keytruda (pembrolizumab) Merck Sharpe & Dohme Corp. 7/15/2019 Patients with hepatocellular carcinoma Cancer
Date of BT Designation Disclosure: 7/15/2019/2019
Approval Date:
Indication: Patients with hepatocellular carcinoma
Category: Cancer
 Avexitide Eiger BioPharmaceuticals, Inc. 6/17/2019 Post-bariatric hypoglycemia (PBH) Other
Date of BT Designation Disclosure: 6/17/2019/2019
Approval Date:
Indication: Post-bariatric hypoglycemia (PBH)
Category: Other
 Aliqopa (copanlisib) Bayer 5/30/2019 Adults patients with relapsed marginal zone lymphoma (MZL) who have received at least 2 prior therapies Cancer
Date of BT Designation Disclosure: 5/30/2019/2019
Approval Date:
Indication: Adults patients with relapsed marginal zone lymphoma (MZL) who have received at least 2 prior therapies
Category: Cancer
 LN-145 Iovance Biotherapeutics, Inc. 5/22/2019 Patients with recurrent, metastatic, or persistent cervical cancer who have progressed on or after chemotherapy Cancer
Date of BT Designation Disclosure: 5/22/2019/2019
Approval Date:
Indication: Patients with recurrent, metastatic, or persistent cervical cancer who have progressed on or after chemotherapy
Category: Cancer
 Enhertu (fam-trastuzumab deruxtecan-nxki) Daiichi Sankyo 5/08/2019 12/20/2019 Adults with unresectable (unable to be removed with surgery) or metastatic (when cancer cells spread to other parts of the body) HER2-positive breast cancer who have received two or more prior anti-HER2-based regimens in the metastatic setting Cancer
Date of BT Designation Disclosure: 5/08/2019/2019
Approval Date: 2019
Indication: Adults with unresectable (unable to be removed with surgery) or metastatic (when cancer cells spread to other parts of the body) HER2-positive breast cancer who have received two or more prior anti-HER2-based regimens in the metastatic setting
Category: Cancer
 SER-109 Seres Therapeutics, Inc. 4/29/2019 Recurrent C. difficile infection Rare Inherited Disorders
Date of BT Designation Disclosure: 4/29/2019/2019
Approval Date:
Indication: Recurrent C. difficile infection
Category: Rare Inherited Disorders
 LJPC-0118 La Jolla Pharmaceutical Company 4/24/2019 Patients with severe malaria Infectious Disease
Date of BT Designation Disclosure: 4/24/2019/2019
Approval Date:
Indication: Patients with severe malaria
Category: Infectious Disease
 elafibranor Genfit 4/19/2019 Patients with primary biliary cholangitis (PBC) Other
Date of BT Designation Disclosure: 4/19/2019/2019
Approval Date:
Indication: Patients with primary biliary cholangitis (PBC)
Category: Other
 Uplinza (inebilizumab-cdon) Viela Bio 4/18/2019 6/11/2020 Patients with neuromyelitis optica spectrum disorder (NMOSD) Rare Inherited Disorders
Date of BT Designation Disclosure: 4/18/2019/2019
Approval Date: 2020
Indication: Patients with neuromyelitis optica spectrum disorder (NMOSD)
Category: Rare Inherited Disorders
 PB2452 PhaseBio Pharmaceuticals 4/08/2019 Reversal agent for the antiplatelet drug ticagrelor Other
Date of BT Designation Disclosure: 4/08/2019/2019
Approval Date:
Indication: Reversal agent for the antiplatelet drug ticagrelor
Category: Other
 Koselugo (Selumetinib) AstraZeneca Plc and Merck & Co., Inc. 4/01/2019 4/10/2020 Treatment of pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN) Rare Inherited Disorders
Date of BT Designation Disclosure: 4/01/2019/2019
Approval Date: 2020
Indication: Treatment of pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN)
Category: Rare Inherited Disorders
 AXS-05 Axsome Therapeutics, Inc. 3/27/2019 Major Depressive Disorder (MDD) Rare Inherited Disorders
Date of BT Designation Disclosure: 3/27/2019/2019
Approval Date:
Indication: Major Depressive Disorder (MDD)
Category: Rare Inherited Disorders
 Tibosovo (ivosidenib) and azacitidine Agios Pharmaceuticals 3/27/2019 newly diagnosed patients with IDH1-mutant acute myeloid leukemia (AML) ≥75 years old or are ineligible for intensive induction chemotherapy Cancer
Date of BT Designation Disclosure: 3/27/2019/2019
Approval Date:
Indication: newly diagnosed patients with IDH1-mutant acute myeloid leukemia (AML) ≥75 years old or are ineligible for intensive induction chemotherapy
Category: Cancer
 Venclexta (venetoclax) Abbvie Inc. 3/15/2019 5/15/2019 In combination with obinutuzumab for treatment of previously untreated patients with chronic lymphocytic leukemia (1L CLL) or small lymphocytic lymphoma (SLL) Cancer
Date of BT Designation Disclosure: 3/15/2019/2019
Approval Date: 2019
Indication: In combination with obinutuzumab for treatment of previously untreated patients with chronic lymphocytic leukemia (1L CLL) or small lymphocytic lymphoma (SLL)
Category: Cancer
 AXS-05 Axsome Therapeutics, Inc. 3/15/2019 Patients with major depressive disorder that is resistant to current treatments Other
Date of BT Designation Disclosure: 3/15/2019/2019
Approval Date:
Indication: Patients with major depressive disorder that is resistant to current treatments
Category: Other
 Gavreto (Pralsentinib) Blueprint Medicines Corporation 2/26/2019 9/04/2020 Patients with RET fusion-positive NSCLC that has progressed on platinum-based chemotherapy Cancer
Date of BT Designation Disclosure: 2/26/2019/2019
Approval Date: 2020
Indication: Patients with RET fusion-positive NSCLC that has progressed on platinum-based chemotherapy
Category: Cancer
 AT-GAA (ATB200 + AT2221) Amicus Therapeutics 2/26/2019 Patients with late onset Pompe disease Rare Inherited Disorders
Date of BT Designation Disclosure: 2/26/2019/2019
Approval Date:
Indication: Patients with late onset Pompe disease
Category: Rare Inherited Disorders
 seladelpar CymaBay Therapeutics 2/20/2019 Patients with early stage primary biliary cholangitis (PBC), in combination with ursodeoxycholic acid (UDCA) Other
Date of BT Designation Disclosure: 2/20/2019/2019
Approval Date:
Indication: Patients with early stage primary biliary cholangitis (PBC), in combination with ursodeoxycholic acid (UDCA)
Category: Other
 CP101 Finch Therapeutics Group, Inc. 2/09/2019 Patients with recurrent Clostridium difficile infection Infectious Disease
Date of BT Designation Disclosure: 2/09/2019/2019
Approval Date:
Indication: Patients with recurrent Clostridium difficile infection
Category: Infectious Disease
 MEDI8897 AstraZeneca 2/06/2019 Prevention of lower respiratory tract infection (LRTI) caused by respiratory syncytial virus (RSV) Other
Date of BT Designation Disclosure: 2/06/2019/2019
Approval Date:
Indication: Prevention of lower respiratory tract infection (LRTI) caused by respiratory syncytial virus (RSV)
Category: Other
 Kadcyla (ado-trastuzumab emtansine) Genentech Inc. 2/05/2019 5/03/2019 Adjuvant treatment of patients with HER2-positive early breast cancer who have residual disease after neoadjuvant taxane and trastuzumab-based treatment Cancer
Date of BT Designation Disclosure: 2/05/2019/2019
Approval Date: 2019
Indication: Adjuvant treatment of patients with HER2-positive early breast cancer who have residual disease after neoadjuvant taxane and trastuzumab-based treatment
Category: Cancer
 umbralisib (TGR-1202) TG Therapeutics 1/28/2019 Adult patients with marginal zone lymphoma (MZL) who did not respond to at least one anti-CD20 therapy Cancer
Date of BT Designation Disclosure: 1/28/2019/2019
Approval Date:
Indication: Adult patients with marginal zone lymphoma (MZL) who did not respond to at least one anti-CD20 therapy
Category: Cancer
 V114 Merck 1/28/2019 Prevention of invasive pneumococcal disease Infectious Disease
Date of BT Designation Disclosure: 1/28/2019/2019
Approval Date:
Indication: Prevention of invasive pneumococcal disease
Category: Infectious Disease
 Brukinsa (zanubrutinib) BeiGene USA, Inc. 1/16/2019 11/14/2019 Patients with mantle cell lymphoma (MCL) who have received at least one prior therapy Cancer
Date of BT Designation Disclosure: 1/16/2019/2019
Approval Date: 2019
Indication: Patients with mantle cell lymphoma (MCL) who have received at least one prior therapy
Category: Cancer
 Adakveo (crizanlizumab-tmca) Novartis Pharmaceuticals Corporation 1/08/2019 11/15/2019 To reduce the frequency of vasoocclusive crises in adults and pediatric patients aged 16 years and older with sickle cell disease Rare Inherited Disorders
Date of BT Designation Disclosure: 1/08/2019/2019
Approval Date: 2019
Indication: To reduce the frequency of vasoocclusive crises in adults and pediatric patients aged 16 years and older with sickle cell disease
Category: Rare Inherited Disorders
 Braftovi (encorafenib) Array BioPharma Inc. 2019 4/08/2020 In combination with cetuximab for the treatment of adult patients with metastatic colorectal cancer (CRC) with a BRAF V600E mutation, detected by an FDA-approved test, after prior therapy Cancer
Date of BT Designation Disclosure: 2019/2019
Approval Date: 2020
Indication: In combination with cetuximab for the treatment of adult patients with metastatic colorectal cancer (CRC) with a BRAF V600E mutation, detected by an FDA-approved test, after prior therapy
Category: Cancer
 TUKYSA (tucatinib) SEATTLE GENETICS INC 2019 4/17/2020 In combination with trastuzumab and capecitabine, for the treatment of adult patients with advanced unresectable or metastatic HER2- positive breast cancer, including patients with brain metastases, who have received one or more prior antiHER2-based regim Cancer
Date of BT Designation Disclosure: 2019/2019
Approval Date: 2020
Indication: In combination with trastuzumab and capecitabine, for the treatment of adult patients with advanced unresectable or metastatic HER2- positive breast cancer, including patients with brain metastases, who have received one or more prior antiHER2-based regim
Category: Cancer
 Enspryng (satralizumab-mwge) Genentech, Inc. 12/20/2018 8/14/2020 Subcutaneous treatment for adults living with anti-aquaporin-4 (AQP4) antibody positive neuromyelitis optica spectrum disorder (NMOSD). Rare Inherited Disorders
Date of BT Designation Disclosure: 12/20/2018/2018
Approval Date: 2020
Indication: Subcutaneous treatment for adults living with anti-aquaporin-4 (AQP4) antibody positive neuromyelitis optica spectrum disorder (NMOSD).
Category: Rare Inherited Disorders
 Olipudase Alfa Sanofi/Genzyme 11/29/2018 Niemann-Pick disease Type B (NPB) Rare Inherited Disorders
Date of BT Designation Disclosure: 11/29/2018/2018
Approval Date:
Indication: Niemann-Pick disease Type B (NPB)
Category: Rare Inherited Disorders
 lonafarnib Eiger BioPharmaceuticals 11/19/2018 11/20/2020 Patients with Hutchinson-Gilford progeria syndrome (HGPS_ Rare Inherited Disorders
Date of BT Designation Disclosure: 11/19/2018/2018
Approval Date: 2020
Indication: Patients with Hutchinson-Gilford progeria syndrome (HGPS_
Category: Rare Inherited Disorders
 Promacta (eltrombopag) Novartis Pharmaceuticals Corp. 11/16/2018 Low platelet counts in people exposed to radiation Other
Date of BT Designation Disclosure: 11/16/2018/2018
Approval Date:
Indication: Low platelet counts in people exposed to radiation
Category: Other
 Emgality (galcanezumab) Eli Lilly & Company 11/15/2018 6/04/2019 Episodic cluster headache in adults Rare Inherited Disorders
Date of BT Designation Disclosure: 11/15/2018/2018
Approval Date: 2019
Indication: Episodic cluster headache in adults
Category: Rare Inherited Disorders
 Adcetris (brentuximab vedotin) Seattle Genetics Inc. 11/15/2018 11/16/2018 Adult patients with previously untreated systemic anaplastic large cell lymphoma or other CD30-expressing peripheral T-cell lymphomas (PTCL) in combination with cyclophosphamide, doxorubicin, and prednisone Cancer
Date of BT Designation Disclosure: 11/15/2018/2018
Approval Date: 2018
Indication: Adult patients with previously untreated systemic anaplastic large cell lymphoma or other CD30-expressing peripheral T-cell lymphomas (PTCL) in combination with cyclophosphamide, doxorubicin, and prednisone
Category: Cancer
 NGX-101 NeuroRx 11/13/2018 Severe Bipolar Depression with Acute Suicidal Ideation & Behavior (ASIB) after initial stabilization with ketamine or other effective therapy Other
Date of BT Designation Disclosure: 11/13/2018/2018
Approval Date:
Indication: Severe Bipolar Depression with Acute Suicidal Ideation & Behavior (ASIB) after initial stabilization with ketamine or other effective therapy
Category: Other
 NRX-101 NeuroRx 11/13/2018 Treatment of Severe Bipolar Depression with Acute Suicidal Ideation & Behavior (ASIB) Other
Date of BT Designation Disclosure: 11/13/2018/2018
Approval Date:
Indication: Treatment of Severe Bipolar Depression with Acute Suicidal Ideation & Behavior (ASIB)
Category: Other
 Jelmyto (mitomycin) UroGen Pharma 10/31/2018 4/15/2020 Treatment of adult patients with lowgrade upper tract urothelial cancer Cancer
Date of BT Designation Disclosure: 10/31/2018/2018
Approval Date: 2020
Indication: Treatment of adult patients with lowgrade upper tract urothelial cancer
Category: Cancer
 Psilocybin therapy COMPASS Pathways 10/23/2018 Treatment-resistant Depression Other
Date of BT Designation Disclosure: 10/23/2018/2018
Approval Date:
Indication: Treatment-resistant Depression
Category: Other
 Myrcludex MYR Pharma 10/23/2018 Chronic hepatitis delta (HDV) infection Infectious Disease
Date of BT Designation Disclosure: 10/23/2018/2018
Approval Date:
Indication: Chronic hepatitis delta (HDV) infection
Category: Infectious Disease
 Psilocybin formulation (Comp 360) Compass Pathways 10/23/2018 Treatment-resistant depression Other
Date of BT Designation Disclosure: 10/23/2018/2018
Approval Date:
Indication: Treatment-resistant depression
Category: Other
 KD025 Kadmon Holdings, Inc. 10/17/2018 Patients with chronic graft-versus-host disease (cGVHD) after failure of two or more lines of systemic therapy Other
Date of BT Designation Disclosure: 10/17/2018/2018
Approval Date:
Indication: Patients with chronic graft-versus-host disease (cGVHD) after failure of two or more lines of systemic therapy
Category: Other
 LOXO-292 Loxo Oncology, Inc. 10/16/2018 Patients with advanced RET fusion-positive thyroid cancer who require systemic therapy, have progressed following prior treatment, and have no acceptable alternative treatment options Cancer
Date of BT Designation Disclosure: 10/16/2018/2018
Approval Date:
Indication: Patients with advanced RET fusion-positive thyroid cancer who require systemic therapy, have progressed following prior treatment, and have no acceptable alternative treatment options
Category: Cancer
 Retevmo (selpercatinib) Loxo Oncology, Inc. 10/15/2018 5/08/2020 Patients with metastatic, RET-fusion-positive non-small-cell lung cancer (NSCLC) who require systemic therapy and progressed following anti PD-L1 therapy or platinum-based chemotherapy plus an anti-PD-1 therapy Cancer
Date of BT Designation Disclosure: 10/15/2018/2018
Approval Date: 2020
Indication: Patients with metastatic, RET-fusion-positive non-small-cell lung cancer (NSCLC) who require systemic therapy and progressed following anti PD-L1 therapy or platinum-based chemotherapy plus an anti-PD-1 therapy
Category: Cancer
 Rubraca (rucaparib) Clovis Oncology 10/03/2018 BRCA1/BRCA2-mutated, metastatic, castration-resistant prostate cancer Cancer
Date of BT Designation Disclosure: 10/03/2018/2018
Approval Date:
Indication: BRCA1/BRCA2-mutated, metastatic, castration-resistant prostate cancer
Category: Cancer
 PF-06482077 Pfizer, Inc. 9/20/2018 Prevention of invasive disease and pneumonia caused by Streptococcus pneumoniae serotypes in the vaccine in adults aged 18 years and older Infectious Disease
Date of BT Designation Disclosure: 9/20/2018/2018
Approval Date:
Indication: Prevention of invasive disease and pneumonia caused by Streptococcus pneumoniae serotypes in the vaccine in adults aged 18 years and older
Category: Infectious Disease
 CA-008 Concentric Analgesics, Inc. 9/11/2018 Patients needing long-lasting, non-opioid pain relief Other
Date of BT Designation Disclosure: 9/11/2018/2018
Approval Date:
Indication: Patients needing long-lasting, non-opioid pain relief
Category: Other
 Tezepelumab AstraZeneca Plc & Amgen Inc. 9/07/2018 Patients with severe asthma, without an eosinophilic phenotype, who are receiving inhaled corticosteroids/long-acting beta2-agonists with or without oral corticosteroids and additional asthma controllers Other
Date of BT Designation Disclosure: 9/07/2018/2018
Approval Date:
Indication: Patients with severe asthma, without an eosinophilic phenotype, who are receiving inhaled corticosteroids/long-acting beta2-agonists with or without oral corticosteroids and additional asthma controllers
Category: Other
 PF-06651600 Pfizer Inc. 9/06/2018 Patients with alopecia areata Other
Date of BT Designation Disclosure: 9/06/2018/2018
Approval Date:
Indication: Patients with alopecia areata
Category: Other
 LOXO-292 Loxo Oncology, Inc. 9/05/2018 Patients with metastatic RET-fusion-positive non-small cell lung cancer; patients with RET-mutant medullary thyroid cancer Cancer
Date of BT Designation Disclosure: 9/05/2018/2018
Approval Date:
Indication: Patients with metastatic RET-fusion-positive non-small cell lung cancer; patients with RET-mutant medullary thyroid cancer
Category: Cancer
 Danyelza (naxitamab-gqgk) Y-mAbs Therapeutics, Inc. 8/23/2018 11/25/2020 In combination with GM-CSF for patients with high risk neuroblastoma refractory to initial therapy or with incomplete response to salvage therapy in patients older than 12 months of age with persistent, refractory disease limited to bone marrow Cancer
Date of BT Designation Disclosure: 8/23/2018/2018
Approval Date: 2020
Indication: In combination with GM-CSF for patients with high risk neuroblastoma refractory to initial therapy or with incomplete response to salvage therapy in patients older than 12 months of age with persistent, refractory disease limited to bone marrow
Category: Cancer
 Xolair (omalizumab) Genentech, Inc. 8/13/2018 9/28/2018 Prevention of severe allergic reactions following accidental exposure to one or more foods in people with allergies Other
Date of BT Designation Disclosure: 8/13/2018/2018
Approval Date: 2018
Indication: Prevention of severe allergic reactions following accidental exposure to one or more foods in people with allergies
Category: Other
 Braftovi (encorafenib) + Mektovi (binimetinib) + Erbitux (cetuximab) Array BioPharma and Eli Lilly and Co. 8/07/2018 Patients with BRAF V600E-mutation metastatic colorectal caner following 1 or 2 prior lines of treatment in the metastatic setting Cancer
Date of BT Designation Disclosure: 8/07/2018/2018
Approval Date:
Indication: Patients with BRAF V600E-mutation metastatic colorectal caner following 1 or 2 prior lines of treatment in the metastatic setting
Category: Cancer
 Quizartinib Daiichi Sankyo Company, Limited 8/01/2018 Adult patients with relapsed/refractory FLT3-ITD acute myeloid leukemia (AML) Cancer
Date of BT Designation Disclosure: 8/01/2018/2018
Approval Date:
Indication: Adult patients with relapsed/refractory FLT3-ITD acute myeloid leukemia (AML)
Category: Cancer
 Keytruda (pembrolizumab) Merck Sharp & Dohme Corp 8/01/2018 9/17/2019 In combination with lenvatinib for the treatment of patients with advanced endometrial carcinoma that is not MSI-H or dMMR, who have disease progression following prior systemic therapy and are not candidates for surgery or radiation Cancer
Date of BT Designation Disclosure: 8/01/2018/2018
Approval Date: 2019
Indication: In combination with lenvatinib for the treatment of patients with advanced endometrial carcinoma that is not MSI-H or dMMR, who have disease progression following prior systemic therapy and are not candidates for surgery or radiation
Category: Cancer
 Lenvima (lenvatinib) Eisai Co. 8/01/2018 9/17/2019 In combination with pembrolizumab for the treatment of patients with previously advanced endometrial carcinoma that is not MSI-H or dMMR, who have disease progression following prior systemic therapy and are not candidates for surgery or radiation Cancer
Date of BT Designation Disclosure: 8/01/2018/2018
Approval Date: 2019
Indication: In combination with pembrolizumab for the treatment of patients with previously advanced endometrial carcinoma that is not MSI-H or dMMR, who have disease progression following prior systemic therapy and are not candidates for surgery or radiation
Category: Cancer
 Avastin (Bevacizumab) GENENTECH INC 7/18/2018 5/29/2020 In combination with atezolizumab, for the treatment of patients with unresectable or metastatic hepatocellular carcinoma who have not received prior systemic therapy Cancer
Date of BT Designation Disclosure: 7/18/2018/2018
Approval Date: 2020
Indication: In combination with atezolizumab, for the treatment of patients with unresectable or metastatic hepatocellular carcinoma who have not received prior systemic therapy
Category: Cancer
 Tecentriq (atezolizumab) Genentech Inc. 7/18/2018 5/29/2020 In combination with bevacizumab, for the treatment of patients with unresectable or metastatic hepatocellular carcinoma who have not received prior systemic therapy Cancer
Date of BT Designation Disclosure: 7/18/2018/2018
Approval Date: 2020
Indication: In combination with bevacizumab, for the treatment of patients with unresectable or metastatic hepatocellular carcinoma who have not received prior systemic therapy
Category: Cancer
 AMT-601 uniQure N.V. 6/28/2018 Patients with severe and moderately severe hemophilia B Rare Inherited Disorders
Date of BT Designation Disclosure: 6/28/2018/2018
Approval Date:
Indication: Patients with severe and moderately severe hemophilia B
Category: Rare Inherited Disorders
 HTX-011 Heron Therapeutics, Inc. 6/21/2018 Postoperative Pain Management Other
Date of BT Designation Disclosure: 6/21/2018/2018
Approval Date:
Indication: Postoperative Pain Management
Category: Other
 Kisqali (ribociclib) Novartis Pharmaceuticals Corp. 6/20/2018 7/18/2018 In combination with fulvestrant for postmenopausal women with HR-positive, HER2-negative advanced or metastatic breast cancer, as initial endocrine-based therapy or following disease progression on endocrine therapy Cancer
Date of BT Designation Disclosure: 6/20/2018/2018
Approval Date: 2018
Indication: In combination with fulvestrant for postmenopausal women with HR-positive, HER2-negative advanced or metastatic breast cancer, as initial endocrine-based therapy or following disease progression on endocrine therapy
Category: Cancer
 Kisqali (ribociclib) Novartis Pharmaceuticals Corp. 6/20/2018 7/18/2018 In combination with an aromatase inhibitor for pre/perimenopausal women with HR-positive, HER2-negative advanced or metastatic breast cancer, as initial endocrine-based therapy Cancer
Date of BT Designation Disclosure: 6/20/2018/2018
Approval Date: 2018
Indication: In combination with an aromatase inhibitor for pre/perimenopausal women with HR-positive, HER2-negative advanced or metastatic breast cancer, as initial endocrine-based therapy
Category: Cancer
 Elzonris (tagraxofusp) Stemline Therapeutics, Inc. 6/15/2018 12/21/2018 Adults and children aged 2 years and older with blastic plasmacytoid dendritic cell neoplasm (BPDCN) Cancer
Date of BT Designation Disclosure: 6/15/2018/2018
Approval Date: 2018
Indication: Adults and children aged 2 years and older with blastic plasmacytoid dendritic cell neoplasm (BPDCN)
Category: Cancer
 Xalkori (crizotinib) Pfizer Inc. 5/29/2018 1/15/2021 Patients with relapsed or refractory systemic anaplastic large cell lymphoma that is anaplastic lymphoma kinase-positive Cancer
Date of BT Designation Disclosure: 5/29/2018/2018
Approval Date: 2021
Indication: Patients with relapsed or refractory systemic anaplastic large cell lymphoma that is anaplastic lymphoma kinase-positive
Category: Cancer
 Xalkori (crizotinib) Pfizer Inc. 5/29/2018 Patients with metastatic non-small cell lung cancer with MET exon 14 alterations with disease progression on or after platinum-based chemotherapy Cancer
Date of BT Designation Disclosure: 5/29/2018/2018
Approval Date:
Indication: Patients with metastatic non-small cell lung cancer with MET exon 14 alterations with disease progression on or after platinum-based chemotherapy
Category: Cancer
 Vyndaquel (tafamidis meglumine) Foldrx Pharmaceuticals, Inc. Sub. Pfizer, Inc. 5/24/2018 5/03/2019 Patients with cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular related hospitalization Other
Date of BT Designation Disclosure: 5/24/2018/2018
Approval Date: 2019
Indication: Patients with cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular related hospitalization
Category: Other
 Tafamidis Pfizer Inc. 5/23/2018 Transthyretin Cardiomyopathy (ATTR-ACT) Cardiovascular
Date of BT Designation Disclosure: 5/23/2018/2018
Approval Date:
Indication: Transthyretin Cardiomyopathy (ATTR-ACT)
Category: Cardiovascular
 Lenti-D Bluebird bio Inc. 5/23/2018 Cerebral adrenoleukodystrophy Rare Inherited Disorders
Date of BT Designation Disclosure: 5/23/2018/2018
Approval Date:
Indication: Cerebral adrenoleukodystrophy
Category: Rare Inherited Disorders
 Pitolisant Harmony Biosciences 5/21/2018 Excessive daytime sleepiness and cataplexy in patients with narcolepsy Other
Date of BT Designation Disclosure: 5/21/2018/2018
Approval Date:
Indication: Excessive daytime sleepiness and cataplexy in patients with narcolepsy
Category: Other
 Trikafta (elexacaftor/ivacaftor/tezacaftor) Vertex Pharmaceuticals Inc. 5/01/2018 10/21/2019 Patients 12 years and older with cystic fibrosis who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene Rare Inherited Disorders
Date of BT Designation Disclosure: 5/01/2018/2018
Approval Date: 2019
Indication: Patients 12 years and older with cystic fibrosis who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
Category: Rare Inherited Disorders
 OMS721 Omeros Corporation 4/26/2018 Patients with high-risk hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), specifically those patents who have persistent TMA despite modification of immunosupressive therapy Other
Date of BT Designation Disclosure: 4/26/2018/2018
Approval Date:
Indication: Patients with high-risk hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), specifically those patents who have persistent TMA despite modification of immunosupressive therapy
Category: Other
 Trumenba (Meningococcal Group B Vaccine) Pfizer Inc. 4/23/2018 Active immunization to prevent invasive meningococcal disease caused by N. meningitidis serogroup B in individuals 1 through 9 years of age
Date of BT Designation Disclosure: 4/23/2018/2018
Approval Date:
Indication: Active immunization to prevent invasive meningococcal disease caused by N. meningitidis serogroup B in individuals 1 through 9 years of age
Category:
 Hemlibra (emicizumab-kxwh) Genentech Inc. 4/17/2018 10/04/2018 Patients with hemophilia A without factor VIII inhibitors Rare Inherited Disorders
Date of BT Designation Disclosure: 4/17/2018/2018
Approval Date: 2018
Indication: Patients with hemophilia A without factor VIII inhibitors
Category: Rare Inherited Disorders
 Ketamine Johnson & Johnson 4/16/2018 Patients with depression Other
Date of BT Designation Disclosure: 4/16/2018/2018
Approval Date:
Indication: Patients with depression
Category: Other
 ALIS (amikacin liposome inhalation suspension) Insmed Inc. 3/29/2018 Adult patients with nontuberculous mycobacterial lung disease caused by Mycobacterium avium complex Other
Date of BT Designation Disclosure: 3/29/2018/2018
Approval Date:
Indication: Adult patients with nontuberculous mycobacterial lung disease caused by Mycobacterium avium complex
Category: Other
 Pedmark (sodium thiosulfate) Fennec Pharmaceuticals Inc. 3/27/2018 Prevention of cisplatin-related ototoxicity in pediatric patients with standard risk hepatoblastoma (SR-HB) Other
Date of BT Designation Disclosure: 3/27/2018/2018
Approval Date:
Indication: Prevention of cisplatin-related ototoxicity in pediatric patients with standard risk hepatoblastoma (SR-HB)
Category: Other
 enfortumab vedotin Astellas Pharma US, Inc. and Seattle Genetics, Inc. 3/26/2018 Patients with locally advanced or metastatic urothelial carcinoma Cancer
Date of BT Designation Disclosure: 3/26/2018/2018
Approval Date:
Indication: Patients with locally advanced or metastatic urothelial carcinoma
Category: Cancer
 Padcev (enfortumab vedotin-ejfv) Astellas Pharma US Inc. 3/26/2018 12/18/2019 Patients with locally advanced or metastatic urothelial cancer who have previously received a PD-1 or PD-L1 inhibitor, and a platinum-containing chemotherapy in the neoadjuvant/adjuvant, locally advanced or metastatic setting Cancer
Date of BT Designation Disclosure: 3/26/2018/2018
Approval Date: 2019
Indication: Patients with locally advanced or metastatic urothelial cancer who have previously received a PD-1 or PD-L1 inhibitor, and a platinum-containing chemotherapy in the neoadjuvant/adjuvant, locally advanced or metastatic setting
Category: Cancer
 PTI-428 Proteostasis Therapeutics 3/16/2018 Cystic Fibrosis for patients with two copies of the F508del mutation — one from their mother and one from their father — who are receiving Vertex’s Orkambi (lumacaftor/ivacaftor) Rare Inherited Disorders
Date of BT Designation Disclosure: 3/16/2018/2018
Approval Date:
Indication: Cystic Fibrosis for patients with two copies of the F508del mutation — one from their mother and one from their father — who are receiving Vertex’s Orkambi (lumacaftor/ivacaftor)
Category: Rare Inherited Disorders
 Balversa (erdafitinib) Janssen Biotech, Inc. 3/15/2018 4/12/2019 Adult patients with locally advanced or metastatic urothelial carcinoma (mUC) that has susceptible FGFR3 or FGFR2 genetic alterations and progressed during at least on line of platinum-containing chemotherapy Cancer
Date of BT Designation Disclosure: 3/15/2018/2018
Approval Date: 2019
Indication: Adult patients with locally advanced or metastatic urothelial carcinoma (mUC) that has susceptible FGFR3 or FGFR2 genetic alterations and progressed during at least on line of platinum-containing chemotherapy
Category: Cancer
 Oxlumo (lumasiran) Alnylam Pharmaceuticals, Inc. 3/12/2018 11/23/2020 Primary Hyperoxaluria Type 1 (PH1) Infectious Disease
Date of BT Designation Disclosure: 3/12/2018/2018
Approval Date: 2020
Indication: Primary Hyperoxaluria Type 1 (PH1)
Category: Infectious Disease
 Orkambi (lumacaftor/ivacaftor) Vertex Pharmaceuticals Inc. 3/12/2018 8/07/2018 Treatment of cystic fibrosis (CF) in patients 2 years and older, homozygous for the F508del-CFTR mutation in the CFTR gene Rare Inherited Disorders
Date of BT Designation Disclosure: 3/12/2018/2018
Approval Date: 2018
Indication: Treatment of cystic fibrosis (CF) in patients 2 years and older, homozygous for the F508del-CFTR mutation in the CFTR gene
Category: Rare Inherited Disorders
 BIVV009 Bioverativ Inc. 3/06/2018 Patients with cold agglutinin disease (CAgD) Other
Date of BT Designation Disclosure: 3/06/2018/2018
Approval Date:
Indication: Patients with cold agglutinin disease (CAgD)
Category: Other
 GC4419 Galera Therapeutics 2/28/2018 Reduction of the duration, severity, and incidence of severe oral mucositis induced by radiation therapy Other
Date of BT Designation Disclosure: 2/28/2018/2018
Approval Date:
Indication: Reduction of the duration, severity, and incidence of severe oral mucositis induced by radiation therapy
Category: Other
 Opdivo (nivolumab) Bristol-Myers Squibb Company 2/16/2018 4/16/2018 In combination with ipilimumab, for treatment of patients with intermediate or poor risk, previously untreated advanced renal cell carcinoma (RCC Cancer
Date of BT Designation Disclosure: 2/16/2018/2018
Approval Date: 2018
Indication: In combination with ipilimumab, for treatment of patients with intermediate or poor risk, previously untreated advanced renal cell carcinoma (RCC
Category: Cancer
 Yervoy (ipilimumab) Bristol-Myers Squibb Company 2/16/2018 4/16/2018 In combination with nivolumab, for treatment of patients with intermediate or poor risk, previously untreated advanced renal cell carcinoma (RCC) Cancer
Date of BT Designation Disclosure: 2/16/2018/2018
Approval Date: 2018
Indication: In combination with nivolumab, for treatment of patients with intermediate or poor risk, previously untreated advanced renal cell carcinoma (RCC)
Category: Cancer
 SPK-8011 Spark Therapeutics Inc. 2/15/2018 Patients with hemophilia A. Rare Inherited Disorders
Date of BT Designation Disclosure: 2/15/2018/2018
Approval Date:
Indication: Patients with hemophilia A.
Category: Rare Inherited Disorders
 Abrocitinib Pfizer Inc. 2/15/2018 Patients with atopic dermatitis Other
Date of BT Designation Disclosure: 2/15/2018/2018
Approval Date:
Indication: Patients with atopic dermatitis
Category: Other
 PF-04965842 Pfizer Inc. 2/14/2018 Patients with moderate-to-severe atopic dermatitis (AD) Other
Date of BT Designation Disclosure: 2/14/2018/2018
Approval Date:
Indication: Patients with moderate-to-severe atopic dermatitis (AD)
Category: Other
 Opdivo (nivolumab) Bristol-Myers Squibb Company 2/10/2018 6/10/2018 Patients 12 years of age and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (mCRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan Cancer
Date of BT Designation Disclosure: 2/10/2018/2018
Approval Date: 2018
Indication: Patients 12 years of age and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (mCRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan
Category: Cancer
 Yervoy (ipilimumab) Bristol-Myers Squibb Company 2/10/2018 7/10/2018 Patients 12 years of age and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (mCRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan Cancer
Date of BT Designation Disclosure: 2/10/2018/2018
Approval Date: 2018
Indication: Patients 12 years of age and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (mCRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan
Category: Cancer
 SAGE-217 Sage Therapeutics 2/07/2018 Patients with major depressive disorder (MDD) Other
Date of BT Designation Disclosure: 2/07/2018/2018
Approval Date:
Indication: Patients with major depressive disorder (MDD)
Category: Other
 ZX008 (fenfluramine) Zogenix, Inc. 2/06/2018 Patients who have seizures associated with Dravet syndrome Other
Date of BT Designation Disclosure: 2/06/2018/2018
Approval Date:
Indication: Patients who have seizures associated with Dravet syndrome
Category: Other
 Onpattro (patisiran) Alnylam Pharmaceuticals Inc. 2/02/2018 8/10/2018 Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults Rare Inherited Disorders
Date of BT Designation Disclosure: 2/02/2018/2018
Approval Date: 2018
Indication: Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
Category: Rare Inherited Disorders
 Balovaptan Roche 1/29/2018 Autism spectrum disorder (ASD) Rare Inherited Disorders
Date of BT Designation Disclosure: 1/29/2018/2018
Approval Date:
Indication: Autism spectrum disorder (ASD)
Category: Rare Inherited Disorders
 Balovaptan (RG7314) Hoffman-La Roche Inc. 1/28/2018 A vasopressin 1a (V1a) receptor antagonist for patients with autism spectrum disorder (ASD) Other
Date of BT Designation Disclosure: 1/28/2018/2018
Approval Date:
Indication: A vasopressin 1a (V1a) receptor antagonist for patients with autism spectrum disorder (ASD)
Category: Other
 Kisquali Femara Co-Pack (letrozole and ribociclib) Novartis Pharmaceuticals Corp. 1/10/2018 2/13/2019 Treatment of pre/perimenopausal women with HR-positive, HER2- negative advanced or metastatic breast cancer Cancer
Date of BT Designation Disclosure: 1/10/2018/2018
Approval Date: 2019
Indication: Treatment of pre/perimenopausal women with HR-positive, HER2- negative advanced or metastatic breast cancer
Category: Cancer
 Upadacitinib (ABT-494) Abbvie 1/09/2018 Adult patients with moderate to severe atopic dermatitis who are candidates for systemic therapy Other
Date of BT Designation Disclosure: 1/09/2018/2018
Approval Date:
Indication: Adult patients with moderate to severe atopic dermatitis who are candidates for systemic therapy
Category: Other
 Lenvima (lenvatinib) in combination with Keytruda (pembrolizumab) Merck and Eisai 1/09/2018 Advanced and/or metastatic renal cell carcinoma (RCC) Cancer
Date of BT Designation Disclosure: 1/09/2018/2018
Approval Date:
Indication: Advanced and/or metastatic renal cell carcinoma (RCC)
Category: Cancer
 Oxbryta (voxelotor) Global Blood Therapeutics, Inc. 1/09/2018 11/25/2019 Sickle cell disease (SCD) in patients 12 years and older Rare Inherited Disorders
Date of BT Designation Disclosure: 1/09/2018/2018
Approval Date: 2019
Indication: Sickle cell disease (SCD) in patients 12 years and older
Category: Rare Inherited Disorders
 Maribavir (SHP620) Shire 1/08/2018 Cytomegalovirus (CMV) infection in transplant patients. Infectious Disease
Date of BT Designation Disclosure: 1/08/2018/2018
Approval Date:
Indication: Cytomegalovirus (CMV) infection in transplant patients.
Category: Infectious Disease
 Promacta (eltrombopag) Novartis Pharmaceuticals Corp. 1/04/2018 11/16/2018 First-line treatment for adults and pediatric patients two years and older with SAA in combination with standard immunosuppressive therapy (IST) Rare Inherited Disorders
Date of BT Designation Disclosure: 1/04/2018/2018
Approval Date: 2018
Indication: First-line treatment for adults and pediatric patients two years and older with SAA in combination with standard immunosuppressive therapy (IST)
Category: Rare Inherited Disorders
 Gamifant (emapalumab-Izsg) Swedish Orphan Biovitrum AB (Publ.) 2018 11/20/2018 Adult and pediatric patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy Rare Inherited Disorders
Date of BT Designation Disclosure: 2018/2018
Approval Date: 2018
Indication: Adult and pediatric patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy
Category: Rare Inherited Disorders
 Venclexta (venetoclax) Abbvie Inc. 2018 6/08/2018 Patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma, with or without 17p deletion, who have received at least one prior therapy Cancer
Date of BT Designation Disclosure: 2018/2018
Approval Date: 2018
Indication: Patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma, with or without 17p deletion, who have received at least one prior therapy
Category: Cancer
 Bavencio (avelumab) EMD Serono Inc. 12/22/2017 5/14/2019 In combination with Inlyta (axitinib) for first-line treatment of patients with advanced renal cell carcinoma (RCC) Cancer
Date of BT Designation Disclosure: 12/22/2017/2017
Approval Date: 2019
Indication: In combination with Inlyta (axitinib) for first-line treatment of patients with advanced renal cell carcinoma (RCC)
Category: Cancer
 Avelumab in combination with Inlyta (axitinib) EMD Serono and Pfizer 12/21/2017 Treatment-naïve patients with advanced renal cell carcinoma (RCC) Cancer
Date of BT Designation Disclosure: 12/21/2017/2017
Approval Date:
Indication: Treatment-naïve patients with advanced renal cell carcinoma (RCC)
Category: Cancer
 Gilenya (fingolimod) Novartis Pharmaceuticals Corp. 12/18/2017 5/11/2018 Patients with relapsing multiple sclerosis (MS) to include pediatric patients 10 years of age and above Rare Inherited Disorders
Date of BT Designation Disclosure: 12/18/2017/2017
Approval Date: 2018
Indication: Patients with relapsing multiple sclerosis (MS) to include pediatric patients 10 years of age and above
Category: Rare Inherited Disorders
 Polivy (polatuzumab vedotin-piiq) Genentech, Inc. 12/10/2017 6/10/2019 In combination with bendamustine and a rituximab product for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma, not otherwise specified, after at least two prior therapies Cancer
Date of BT Designation Disclosure: 12/10/2017/2017
Approval Date: 2019
Indication: In combination with bendamustine and a rituximab product for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma, not otherwise specified, after at least two prior therapies
Category: Cancer
 Keytruda (pembrolizumab) Merck Sharpe & Dohme Corp. 12/10/2017 4/19/2019 In combination with axitinib for the first-line treatment of patients with advanced renal cell carcinoma (RCC) Cancer
Date of BT Designation Disclosure: 12/10/2017/2017
Approval Date: 2019
Indication: In combination with axitinib for the first-line treatment of patients with advanced renal cell carcinoma (RCC)
Category: Cancer
 Oxervate (cenegermin-bkbj ophthalmic solution) Dompe Farmaceutici SpA 11/30/2017 8/22/2018 Patients with neurotrophic keratitis (NK) Other
Date of BT Designation Disclosure: 11/30/2017/2017
Approval Date: 2018
Indication: Patients with neurotrophic keratitis (NK)
Category: Other
 Idecabtagene vicleucel Bristol-Myers Squibb Company 11/15/2017 Adult patients with relapsed or refractory multiple myeloma Cancer
Date of BT Designation Disclosure: 11/15/2017/2017
Approval Date:
Indication: Adult patients with relapsed or refractory multiple myeloma
Category: Cancer
 bb2121 Bristol Myers Squibb and Bluebird Bio 11/04/2017 Patients with relapsed or refractory multiple myeloma Cancer
Date of BT Designation Disclosure: 11/04/2017/2017
Approval Date:
Indication: Patients with relapsed or refractory multiple myeloma
Category: Cancer
 Blenrep (BELANTAMAB MAFODOTIN-BLMF) GlaxoSmithKline Intellectual Property Development LTD England 11/02/2017 8/05/2020 Treatment of adults with relapsed or refractory multiple myeloma who have received at least four prior therapies including an anti-CD38 monoclonal antibody, a proteasome inhibitor, and an immunomodulatory agent Cancer
Date of BT Designation Disclosure: 11/02/2017/2017
Approval Date: 2020
Indication: Treatment of adults with relapsed or refractory multiple myeloma who have received at least four prior therapies including an anti-CD38 monoclonal antibody, a proteasome inhibitor, and an immunomodulatory agent
Category: Cancer
 Valoctocogene Roxaparvovec BioMarin 10/26/2017 Hemophilia A Rare Inherited Disorders
Date of BT Designation Disclosure: 10/26/2017/2017
Approval Date:
Indication: Hemophilia A
Category: Rare Inherited Disorders
 Poteligeo (mogamulizumab-kpkc) Kyowa Kirin, Inc. 10/25/2017 8/08/2018 Adult patients Sézary syndrome (SS) after at least one prior systemic therapy Cancer
Date of BT Designation Disclosure: 10/25/2017/2017
Approval Date: 2018
Indication: Adult patients Sézary syndrome (SS) after at least one prior systemic therapy
Category: Cancer
 Venclexta (venetoclax) Abbvie Inc. 10/25/2017 11/21/2018 In combination with azacitidine, decitabine, or low-dose cytarabine for adults aged 75 and older with treatment-naive acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy Cancer
Date of BT Designation Disclosure: 10/25/2017/2017
Approval Date: 2018
Indication: In combination with azacitidine, decitabine, or low-dose cytarabine for adults aged 75 and older with treatment-naive acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy
Category: Cancer
 Monjuvi MorpoSys 10/23/2017 7/31/2020 Relapsed/Refractory DLBCL who are not eligible for high-dose chemotherapy and autologous stem-cell transplantation Cancer
Date of BT Designation Disclosure: 10/23/2017/2017
Approval Date: 2020
Indication: Relapsed/Refractory DLBCL who are not eligible for high-dose chemotherapy and autologous stem-cell transplantation
Category: Cancer
 Tafinlar (dabrafenib) Novartis Pharmaceuticals Corp. 10/23/2017 4/30/2018 In combination with trametinib for the adjuvant treatment of patients with stage III melanoma with a BRAF V600E or V600K mutation, as detected by and FDA-approved test, and involvement of lymph node(s), following complete resection Cancer
Date of BT Designation Disclosure: 10/23/2017/2017
Approval Date: 2018
Indication: In combination with trametinib for the adjuvant treatment of patients with stage III melanoma with a BRAF V600E or V600K mutation, as detected by and FDA-approved test, and involvement of lymph node(s), following complete resection
Category: Cancer
 Mekinist (trametinib) Novartis Pharmaceuticals Corp. 10/23/2017 4/30/2018 In combination with dabrafenib for the adjuvant treatment of patients with stage III melanoma with a BRAF V600E or V600K mutation, as detected by and FDA-approved test, and involvement of lymph node(s), following complete resection Cancer
Date of BT Designation Disclosure: 10/23/2017/2017
Approval Date: 2018
Indication: In combination with dabrafenib for the adjuvant treatment of patients with stage III melanoma with a BRAF V600E or V600K mutation, as detected by and FDA-approved test, and involvement of lymph node(s), following complete resection
Category: Cancer
 Mekinist (trametinib) Novartis Pharmaceuticals Corp. 10/23/2017 5/04/2018 In combination with dabrafenib for the treatment of patients with locally advanced or metastatic anaplastic thyroid cancer (ATC) with BRAF V600E mutation and with no satisfactory locoregional treatment options Cancer
Date of BT Designation Disclosure: 10/23/2017/2017
Approval Date: 2018
Indication: In combination with dabrafenib for the treatment of patients with locally advanced or metastatic anaplastic thyroid cancer (ATC) with BRAF V600E mutation and with no satisfactory locoregional treatment options
Category: Cancer
 Tafinlar (dabrafenib) Novartis Pharmaceuticals Corp. 10/23/2017 5/04/2018 In combination with trametinib for the treatment of patients with locally advanced or metastatic anaplastic thyroid cancer (ATC) with BRAF V600E mutation and with no satisfactory locoregional treatment options Cancer
Date of BT Designation Disclosure: 10/23/2017/2017
Approval Date: 2018
Indication: In combination with trametinib for the treatment of patients with locally advanced or metastatic anaplastic thyroid cancer (ATC) with BRAF V600E mutation and with no satisfactory locoregional treatment options
Category: Cancer
 Opdivo (nivolumab) Bristol-Myers Squibb Company 10/17/2017 12/20/2017 Adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection Cancer
Date of BT Designation Disclosure: 10/17/2017/2017
Approval Date: 2017
Indication: Adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection
Category: Cancer
 Adcetris (brentuximab vedotin) Seattle Genetics Inc. 10/15/2017 3/20/2018 Treatment for adult patients with previously untreated Stage III or IV classical Hodgkin Lymphoma, in combination with chemotherapy Cancer
Date of BT Designation Disclosure: 10/15/2017/2017
Approval Date: 2018
Indication: Treatment for adult patients with previously untreated Stage III or IV classical Hodgkin Lymphoma, in combination with chemotherapy
Category: Cancer
 DAS181 Ansun BioPharma 10/10/2017 Treatment of lower respiratory tract parainfluenza virus (PIV) infection in immunocompromised patients Infectious Disease
Date of BT Designation Disclosure: 10/10/2017/2017
Approval Date:
Indication: Treatment of lower respiratory tract parainfluenza virus (PIV) infection in immunocompromised patients
Category: Infectious Disease
 Tagrisso (osimertinib) AstraZeneca Pharmaceuticals LP 10/09/2017 4/18/2018 First-line treatment of patients with metastatic NSCLC whose tumors have EGFR exon 19 deletions of L858R substitution mutations, as detected by an FDA approved test Cancer
Date of BT Designation Disclosure: 10/09/2017/2017
Approval Date: 2018
Indication: First-line treatment of patients with metastatic NSCLC whose tumors have EGFR exon 19 deletions of L858R substitution mutations, as detected by an FDA approved test
Category: Cancer
 Adcetris (brentuximab vedotin) Seattle Genetics Inc. 10/02/2017 11/09/2017 Patients with primary cutaneous anaplastic large cell lymphoma or CD30-expressing mycosis fungoides who have received prior systemic therapy Cancer
Date of BT Designation Disclosure: 10/02/2017/2017
Approval Date: 2017
Indication: Patients with primary cutaneous anaplastic large cell lymphoma or CD30-expressing mycosis fungoides who have received prior systemic therapy
Category: Cancer
 Libtayo (cemiplimab-rwlc) Regeneron Pharmaceuticals Inc. 9/08/2017 9/28/2018 Patients with metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation Cancer
Date of BT Designation Disclosure: 9/08/2017/2017
Approval Date: 2018
Indication: Patients with metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation
Category: Cancer
 EB-101 Abeona Therapeutics Inc 8/30/2017 Recessive Dystrophic Epidermolysis Bullosa (RDEB) Other
Date of BT Designation Disclosure: 8/30/2017/2017
Approval Date:
Indication: Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Category: Other
 Trastuzumab Deruxtecan (DS-8201) Daiichi Sankyo 8/29/2017 HER2-positive, locally advanced, or metastatic breast cancer who have been treated with trastuzumab (Herceptin) and pertuzumab (Perjeta) and have disease progression after ado-trastuzumab emtansine (T-DM1; Kadcyla) Cancer
Date of BT Designation Disclosure: 8/29/2017/2017
Approval Date:
Indication: HER2-positive, locally advanced, or metastatic breast cancer who have been treated with trastuzumab (Herceptin) and pertuzumab (Perjeta) and have disease progression after ado-trastuzumab emtansine (T-DM1; Kadcyla)
Category: Cancer
 Methylenedioxymethamphetamine (MDMA) MAPS 8/28/2017 PTSD Other
Date of BT Designation Disclosure: 8/28/2017/2017
Approval Date:
Indication: PTSD
Category: Other
 Poteligeo (mogamulizumab-kpkc) Kyowa Kirin, Inc. 8/25/2017 8/08/2018 Adult patients with relapsed or refractory mycosis fungoides (MF) or Sezary syndrome after at least one prior systemic therapy Cancer
Date of BT Designation Disclosure: 8/25/2017/2017
Approval Date: 2018
Indication: Adult patients with relapsed or refractory mycosis fungoides (MF) or Sezary syndrome after at least one prior systemic therapy
Category: Cancer
 Zelboraf (vemurafenib) Hoffmann La Roche Inc. 8/07/2017 11/06/2017 Patients with Erdheim-Chester disease (ECD) with a BRAF V600 mutation Other
Date of BT Designation Disclosure: 8/07/2017/2017
Approval Date: 2017
Indication: Patients with Erdheim-Chester disease (ECD) with a BRAF V600 mutation
Category: Other
 Venetoclax Abbvie/Roche 8/01/2017 Elderly patients with previously untreated Acute Myeloid Leukemia (AML) who are ineligible for intensive chemotherapy Cancer
Date of BT Designation Disclosure: 8/01/2017/2017
Approval Date:
Indication: Elderly patients with previously untreated Acute Myeloid Leukemia (AML) who are ineligible for intensive chemotherapy
Category: Cancer
 Acalabrutinib AstraZeneca 8/01/2017 Mantle cell lymphoma for patients who have received at least one prior line of therapy Cancer
Date of BT Designation Disclosure: 8/01/2017/2017
Approval Date:
Indication: Mantle cell lymphoma for patients who have received at least one prior line of therapy
Category: Cancer
 Calquence (acalabrutinib) AstraZeneca UK Ltd. 8/01/2017 10/31/2017 Adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy Cancer
Date of BT Designation Disclosure: 8/01/2017/2017
Approval Date: 2017
Indication: Adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy
Category: Cancer
 Imfinzi (durvalumab) AstraZeneca UK Ltd 7/31/2017 2/16/2018 Patients with unresectable Stage III non-small cell lung cancer (NSCLC) whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy Cancer
Date of BT Designation Disclosure: 7/31/2017/2017
Approval Date: 2018
Indication: Patients with unresectable Stage III non-small cell lung cancer (NSCLC) whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy
Category: Cancer
 Keytruda (pembrolizumab) Merck Sharp & Dohme Corp 7/01/2017 12/19/2018 Adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC) Cancer
Date of BT Designation Disclosure: 7/01/2017/2017
Approval Date: 2018
Indication: Adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC)
Category: Cancer
 Korsuva (difelikefalin) Cara Therapeutics Inc. 6/22/2017 Chronic kidney disease, associated pruritius in hemodialysis patients Other
Date of BT Designation Disclosure: 6/22/2017/2017
Approval Date:
Indication: Chronic kidney disease, associated pruritius in hemodialysis patients
Category: Other
 OMS721 Omeros 6/13/2017 Immunoglobulin A (IgA) nephropathy Other
Date of BT Designation Disclosure: 6/13/2017/2017
Approval Date:
Indication: Immunoglobulin A (IgA) nephropathy
Category: Other
 Burtomab Y-mAbs Therapeutics, Inc. 6/07/2017 Pediatric patients with relapsed or refractory neuroblastoma with central nervous system or leptomeningeal metastasis Cancer
Date of BT Designation Disclosure: 6/07/2017/2017
Approval Date:
Indication: Pediatric patients with relapsed or refractory neuroblastoma with central nervous system or leptomeningeal metastasis
Category: Cancer
 Ayvakit (avapritinib) Blueprint Medicines Corporation 6/01/2017 1/09/2020 Patients with unresectable or metastatic GIST harboring the PDGFRα D842V mutation Cancer
Date of BT Designation Disclosure: 6/01/2017/2017
Approval Date: 2020
Indication: Patients with unresectable or metastatic GIST harboring the PDGFRα D842V mutation
Category: Cancer
 Givlaari (givosiran) Alnylam 5/30/2017 11/20/2019 Adult patients with acute hepatic porphyria (AHP) Rare Inherited Disorders
Date of BT Designation Disclosure: 5/30/2017/2017
Approval Date: 2019
Indication: Adult patients with acute hepatic porphyria (AHP)
Category: Rare Inherited Disorders
 Zemdri (plazomicin) Achaogen 5/23/2017 Antibacterials addressing multi-drug resistant (MDR) gram-negative infections Other
Date of BT Designation Disclosure: 5/23/2017/2017
Approval Date:
Indication: Antibacterials addressing multi-drug resistant (MDR) gram-negative infections
Category: Other
 GMI-1271 GlycoMimetics 5/17/2017 Adult Relapsed/Refractory Acute Myeloid Leukemia Cancer
Date of BT Designation Disclosure: 5/17/2017/2017
Approval Date:
Indication: Adult Relapsed/Refractory Acute Myeloid Leukemia
Category: Cancer
 Rozlytrek (entrectinib) Genentech, Inc. 5/15/2017 8/15/2019 Adult and pediatric patients 12 years of age and older with solid tumors that have a NTRK gene fusion without a known acquired resistance mutation, are metastatic, and have progressed following treatment or have no alternative treatment option Cancer
Date of BT Designation Disclosure: 5/15/2017/2017
Approval Date: 2019
Indication: Adult and pediatric patients 12 years of age and older with solid tumors that have a NTRK gene fusion without a known acquired resistance mutation, are metastatic, and have progressed following treatment or have no alternative treatment option
Category: Cancer
 Ribaxamase (SYN-004) Synthetic Biologics, Inc. 5/11/2017 prevention of Clostridium difficile infection Infectious Disease
Date of BT Designation Disclosure: 5/11/2017/2017
Approval Date:
Indication: prevention of Clostridium difficile infection
Category: Infectious Disease
 Vonapanitase Proteon Therapeutics, Inc. 5/10/2017 Increasing arteriovenous fistula secondary patency (i.e., survival of the fistula without abandonment) and use for hemodialysis in patients on or expected to initiate hemodialysis Other
Date of BT Designation Disclosure: 5/10/2017/2017
Approval Date:
Indication: Increasing arteriovenous fistula secondary patency (i.e., survival of the fistula without abandonment) and use for hemodialysis in patients on or expected to initiate hemodialysis
Category: Other
 Lorbrena (lorlatinib) Pfizer Inc. 4/27/2017 11/02/2018 Patients with ALK-positive metastatic NSCLC whose disease has progressed on crizotinib and at least one other ALK inhibitor for metastatic disease or alectinib or ceritinib Cancer
Date of BT Designation Disclosure: 4/27/2017/2017
Approval Date: 2018
Indication: Patients with ALK-positive metastatic NSCLC whose disease has progressed on crizotinib and at least one other ALK inhibitor for metastatic disease or alectinib or ceritinib
Category: Cancer
 Kymriah (tisagenlecleucel) Novartis Pharmaceutical Corp. 4/18/2017 Patients with large B-cell lymphoma Cancer
Date of BT Designation Disclosure: 4/18/2017/2017
Approval Date:
Indication: Patients with large B-cell lymphoma
Category: Cancer
 Kymriah (CTL019) Novartis 4/18/2017 Adult patients with relapsed and refractory diffuse large B-cell lymphoma, who have failed two or more prior therapies Cancer
Date of BT Designation Disclosure: 4/18/2017/2017
Approval Date:
Indication: Adult patients with relapsed and refractory diffuse large B-cell lymphoma, who have failed two or more prior therapies
Category: Cancer
 RVT-802 Enzyvant 4/17/2017 Complete DiGeorge Syndrome (cDGS) Rare Inherited Disorders
Date of BT Designation Disclosure: 4/17/2017/2017
Approval Date:
Indication: Complete DiGeorge Syndrome (cDGS)
Category: Rare Inherited Disorders
 Evinacumab Regeneron 4/07/2017 Homozygous familial hypercholesterolemia (HOFH) Rare Inherited Disorders
Date of BT Designation Disclosure: 4/07/2017/2017
Approval Date:
Indication: Homozygous familial hypercholesterolemia (HOFH)
Category: Rare Inherited Disorders
 Rituximab (Rituxan) Genentech Inc. 3/24/2017 6/07/2018 Patients with moderate to severe pemphigus vulgaris (PV) Rare Inherited Disorders
Date of BT Designation Disclosure: 3/24/2017/2017
Approval Date: 2018
Indication: Patients with moderate to severe pemphigus vulgaris (PV)
Category: Rare Inherited Disorders
 Prevymis (letermovir) Merck Sharp & Dohme Corp. 2/27/2017 11/08/2017 Prophylaxis of cytomegalovirus (CMV) infection and disease in adult CMV-seropositive recipients [R+] of an allogeneic hematopoietic stem cell transplant (HSCT) Infectious Disease
Date of BT Designation Disclosure: 2/27/2017/2017
Approval Date: 2017
Indication: Prophylaxis of cytomegalovirus (CMV) infection and disease in adult CMV-seropositive recipients [R+] of an allogeneic hematopoietic stem cell transplant (HSCT)
Category: Infectious Disease
 Toca 511 & Toca FC Tocagen 2/23/2017 Recurrent high grade glioma Cancer
Date of BT Designation Disclosure: 2/23/2017/2017
Approval Date:
Indication: Recurrent high grade glioma
Category: Cancer
 AMT-060 uniQure 1/20/2017 Hemophilia B Rare Inherited Disorders
Date of BT Designation Disclosure: 1/20/2017/2017
Approval Date:
Indication: Hemophilia B
Category: Rare Inherited Disorders
 Nuplazid (pimavanserin) Acadia Pharmaceuticals Inc. 2017 Patients with dementia-related psychosis (DRP) Other
Date of BT Designation Disclosure: 2017/2017
Approval Date:
Indication: Patients with dementia-related psychosis (DRP)
Category: Other
 Keytruda (pembrolizumab) Merck Sharp & Dohme Corp. 2017 6/13/2018 Adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after two or more prior lines of therapy Cancer
Date of BT Designation Disclosure: 2017/2017
Approval Date: 2018
Indication: Adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after two or more prior lines of therapy
Category: Cancer
 Ibrance (palbociclib) Pfizer Inc. 2017 4/04/2019 In combination with an aromatase inhibitor, or in combination with fulvestrant, for treatment of male patients with advanced or metastatic breast cancer Cancer
Date of BT Designation Disclosure: 2017/2017
Approval Date: 2019
Indication: In combination with an aromatase inhibitor, or in combination with fulvestrant, for treatment of male patients with advanced or metastatic breast cancer
Category: Cancer
 Keytruda (pembrolizumab) Merck Sharp & Dohme Corp. 2017 5/18/2017 Patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or within 12 months of neo- or neo-adjuvant treatment with platinum-containing chemotherapy Cancer
Date of BT Designation Disclosure: 2017/2017
Approval Date: 2017
Indication: Patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or within 12 months of neo- or neo-adjuvant treatment with platinum-containing chemotherapy
Category: Cancer
 Keytruda (pembrolizumab) Merck Sharp & Dohme Corp. 2017 5/23/2017 Adult and pediatric patients with unresectable or metastatic MSI-H or mismatch repair deficient solid tumors that have progressed following prior treatment or metastatic, MSI-H or mistmatch repair colorectal cancer that has progressed following treatment Cancer
Date of BT Designation Disclosure: 2017/2017
Approval Date: 2017
Indication: Adult and pediatric patients with unresectable or metastatic MSI-H or mismatch repair deficient solid tumors that have progressed following prior treatment or metastatic, MSI-H or mistmatch repair colorectal cancer that has progressed following treatment
Category: Cancer
 Zykadia (ceritinib) Novartis Pharmaceuticals Corp. 2017 5/26/2017 Patients with metastatic non-small cell lung cancer (NSCLC) whose tumors are anaplastic lymphoma kinase (ALK)-positive as detected by an FDA approved test Cancer
Date of BT Designation Disclosure: 2017/2017
Approval Date: 2017
Indication: Patients with metastatic non-small cell lung cancer (NSCLC) whose tumors are anaplastic lymphoma kinase (ALK)-positive as detected by an FDA approved test
Category: Cancer
 Ibrance (palbociclib) Pfizer Inc. 2017 3/31/2017 Postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer, in combination with an aromatase inhibitor as initial endocrine-based therapy Cancer
Date of BT Designation Disclosure: 2017/2017
Approval Date: 2017
Indication: Postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer, in combination with an aromatase inhibitor as initial endocrine-based therapy
Category: Cancer
 Mekinist (trametinib) Novartis Pharmaceuticals Corp. 2017 6/22/2017 Patients with metastatic non-small cell lung cancer (NSCLC) with BRAF V600E mutation as detected by and FDA-approved test Cancer
Date of BT Designation Disclosure: 2017/2017
Approval Date: 2017
Indication: Patients with metastatic non-small cell lung cancer (NSCLC) with BRAF V600E mutation as detected by and FDA-approved test
Category: Cancer
 Besponsa (inotuzumab ozogamicin) Wyeth Pharmaceuticals LLC 2017 8/17/2017 Adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) Cancer
Date of BT Designation Disclosure: 2017/2017
Approval Date: 2017
Indication: Adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL)
Category: Cancer
 Liso-cel (Lisocabtagene maraleucel) Juno Therapeutics/Celgene 12/20/2016 Relapsed/refractory (r/r) aggressive large B-cell NHL, including diffuse large B-cell lymphoma (DLBCL), not otherwise specified (de novo or transformed from indolent lymphoma), Primary Mediastinal B-cell Lymphoma (PMBCL) or Grade 3B Follicular Lymphoma Cancer
Date of BT Designation Disclosure: 12/20/2016/2016
Approval Date:
Indication: Relapsed/refractory (r/r) aggressive large B-cell NHL, including diffuse large B-cell lymphoma (DLBCL), not otherwise specified (de novo or transformed from indolent lymphoma), Primary Mediastinal B-cell Lymphoma (PMBCL) or Grade 3B Follicular Lymphoma
Category: Cancer
 Tonmya (TNX-102 SL) Tonix Pharmaceuticals 12/19/2016 PTSD Other
Date of BT Designation Disclosure: 12/19/2016/2016
Approval Date:
Indication: PTSD
Category: Other
 Neridronic acid Grünenthal and Abiogen Pharma 12/16/2016 Complex regional pain syndrome (CRPS) Rare Inherited Disorders
Date of BT Designation Disclosure: 12/16/2016/2016
Approval Date:
Indication: Complex regional pain syndrome (CRPS)
Category: Rare Inherited Disorders
 Advantage Arrest™ Silver Diamine Fluoride 38% Elevate Oral Care 11/03/2016 Arrest of tooth decay in children and adults Other
Date of BT Designation Disclosure: 11/03/2016/2016
Approval Date:
Indication: Arrest of tooth decay in children and adults
Category: Other
 Zejula (niraparib) Tesaro Inc. 10/14/2016 3/27/2017 Maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy Cancer
Date of BT Designation Disclosure: 10/14/2016/2016
Approval Date: 2017
Indication: Maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy
Category: Cancer
 omidubicel Gamida Cell Ltd. 10/11/2016 Patients with high-risk blood cancers who do not have a suitable matched donor Cancer
Date of BT Designation Disclosure: 10/11/2016/2016
Approval Date:
Indication: Patients with high-risk blood cancers who do not have a suitable matched donor
Category: Cancer
 Omidubicel (formerly known as NiCord®) Gamida Cell (Emmes) 10/11/2016 Treatment opportunity for patients who need an allogeneic bone marrow transplant. Cellular therapy in Phase 3 development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution Cancer
Date of BT Designation Disclosure: 10/11/2016/2016
Approval Date:
Indication: Treatment opportunity for patients who need an allogeneic bone marrow transplant. Cellular therapy in Phase 3 development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution
Category: Cancer
 Elbit Imaging Gamida Cell 10/10/2016 Nicord Cancer
Date of BT Designation Disclosure: 10/10/2016/2016
Approval Date:
Indication: Nicord
Category: Cancer
 Actemra (tocilizumab) Genentech Inc. 10/05/2016 5/22/2017 Adult patients with giant cell arteritis (GCA) Rare Inherited Disorders
Date of BT Designation Disclosure: 10/05/2016/2016
Approval Date: 2017
Indication: Adult patients with giant cell arteritis (GCA)
Category: Rare Inherited Disorders
 Alecensa (alectinib) Roche 10/04/2016 Adult patients with advanced ALK-positive non-small cell lung cancer (NSCLC) who have not received prior treatment with an ALK inhibitor Cancer
Date of BT Designation Disclosure: 10/04/2016/2016
Approval Date:
Indication: Adult patients with advanced ALK-positive non-small cell lung cancer (NSCLC) who have not received prior treatment with an ALK inhibitor
Category: Cancer
 Dupixent (Dupilumab) Regeneron Pharmaceuticals Inc. 10/01/2016 3/11/2019 Patients 12 to less than 18 years of age with moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable Other
Date of BT Designation Disclosure: 10/01/2016/2016
Approval Date: 2019
Indication: Patients 12 to less than 18 years of age with moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable
Category: Other
 ABT-530 Abbvie 9/30/2016 genotype 1 HCV patients who have failed to respond to a previous therapy of direct acting antivirals (or DAAs) Infectious Disease
Date of BT Designation Disclosure: 9/30/2016/2016
Approval Date:
Indication: genotype 1 HCV patients who have failed to respond to a previous therapy of direct acting antivirals (or DAAs)
Category: Infectious Disease
 Mavyret (glecaprevir & pibrentasvir) AbbVie Inc. 9/30/2016 8/03/2017 Patients with chronic hepatitis C virus (HCV) genotype (GT) 1, 2, 3, 4, 5, or 6 infection without cirrhosis or with compensated cirrhosis; and also for patients with HCV GT1 infection previously treated w/ HCV NS5A or NS3/4A inhibitor but not both Infectious Disease
Date of BT Designation Disclosure: 9/30/2016/2016
Approval Date: 2017
Indication: Patients with chronic hepatitis C virus (HCV) genotype (GT) 1, 2, 3, 4, 5, or 6 infection without cirrhosis or with compensated cirrhosis; and also for patients with HCV GT1 infection previously treated w/ HCV NS5A or NS3/4A inhibitor but not both
Category: Infectious Disease
 Zulresso (Brexanolone) Sage Therapeutics Inc. 9/06/2016 3/19/2019 Postpartum depression (PPD) Other
Date of BT Designation Disclosure: 9/06/2016/2016
Approval Date: 2019
Indication: Postpartum depression (PPD)
Category: Other
 SL-401 Stemline Therapeutics 8/23/2016 Treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) targeting the interleukin-3 receptor (CD123) Rare Inherited Disorders
Date of BT Designation Disclosure: 8/23/2016/2016
Approval Date:
Indication: Treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) targeting the interleukin-3 receptor (CD123)
Category: Rare Inherited Disorders
 Esketamine Janssen 8/16/2016 7/31/2020 Major depressive disorder with imminent risk for suicide Other
Date of BT Designation Disclosure: 8/16/2016/2016
Approval Date: 2020
Indication: Major depressive disorder with imminent risk for suicide
Category: Other
 Kisqali (ribociclib) Novartis Pharmaceuticals Corp. 8/03/2016 3/13/2017 Postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer as initial endocrine-based therapy Cancer
Date of BT Designation Disclosure: 8/03/2016/2016
Approval Date: 2017
Indication: Postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer as initial endocrine-based therapy
Category: Cancer
 Kisqali Femara Co-Pack (letrozole and ribociclib) Novartis Pharmaceuticals Corp. 8/02/2016 5/04/2017 Postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced metastatic breast cancer Cancer
Date of BT Designation Disclosure: 8/02/2016/2016
Approval Date: 2017
Indication: Postmenopausal women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced metastatic breast cancer
Category: Cancer
 Pracinostat MEI Pharma 8/01/2016 In combination with azacitidine of patients with newly diagnosed acute myeloid leukemia unfit for intensive chemotherapy Cancer
Date of BT Designation Disclosure: 8/01/2016/2016
Approval Date:
Indication: In combination with azacitidine of patients with newly diagnosed acute myeloid leukemia unfit for intensive chemotherapy
Category: Cancer
 Darzalex (daratumumab) Janssen Biotech Inc. 7/26/2016 11/21/2016 Patients with multiple myeloma who have received at least one prior therapy, in combination with bortezomib and dexamethasone Cancer
Date of BT Designation Disclosure: 7/26/2016/2016
Approval Date: 2016
Indication: Patients with multiple myeloma who have received at least one prior therapy, in combination with bortezomib and dexamethasone
Category: Cancer
 V920 Merck 7/25/2016 Ebola Zaire Infectious Disease
Date of BT Designation Disclosure: 7/25/2016/2016
Approval Date:
Indication: Ebola Zaire
Category: Infectious Disease
 SPK-9001 Pfizer/Spark 7/21/2016 Hemophilia B. Rare Inherited Disorders
Date of BT Designation Disclosure: 7/21/2016/2016
Approval Date:
Indication: Hemophilia B.
Category: Rare Inherited Disorders
 Zolgensma (onasemnogene abeparvovec-xioi) AveXis 7/20/2016 5/24/2019 Spinal muscular atrophy (SMA) Type 1 in children less than two years old Other