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 August 31, 2021, the FDA has approved 251* breakthrough therapy designated products and lists that there have been 1182 total requests for the designation with 458** designations granted.
Trade Name (Agent)
Sponsor
Date of BT Designation Disclosure
Approval Date
Indication
Category
Trade Name (Agent): Xalkori (crizotinib)
Sponsor:
Date of BT Designation Disclosure: 5/29/2018
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
Trade Name (Agent): Xalkori (crizotinib)
Sponsor:
Date of BT Designation Disclosure: 5/29/2018
Approval Date: 1/14/2021
Indication: Patients with relapsed or refractory systemic anaplastic large cell lymphoma that is anaplastic lymphoma kinase-positive
Category: Cancer
Trade Name (Agent): Venclexta (venetoclax) + Rituximab
Sponsor:
Date of BT Designation Disclosure: 1/20/2016
Approval Date: 6/08/2018
Indication: Patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), with or without 17p deletion, who have received at least one prior therapy
Category: Cancer
Trade Name (Agent): HTX-011
Sponsor:
Date of BT Designation Disclosure: 6/21/2018
Indication: Postoperative Pain Management
Category: Other
Trade Name (Agent): Zemdri (plazomicin)
Sponsor:
Date of BT Designation Disclosure: 5/23/2017
Indication: Antibacterials addressing multi-drug resistant (MDR) gram-negative infections
Category: Other
Trade Name (Agent): AMT-601
Sponsor: uniQure, uniQure N.V.
Date of BT Designation Disclosure: 6/28/2018
Indication: Patients with severe and moderately severe hemophilia B
Category: Rare Inherited Disorders
Trade Name (Agent): Kisqali (ribociclib)
Sponsor:
Date of BT Designation Disclosure: 6/20/2018
Approval Date: 7/18/2018
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
Trade Name (Agent): Trumenba (Meningococcal Group B Vaccine)
Sponsor:
Date of BT Designation Disclosure: 3/20/2014
Approval Date: 10/29/2014
Indication: Active immunization to prevent invasive meningococcal disease caused by N. meningitidis serogroup B in individuals 10 through 25 years of age
Category: Infectious Disease
Trade Name (Agent): Bexsero (Meningococcal Group B Vaccine)
Sponsor:
Date of BT Designation Disclosure: 4/07/2014
Approval Date: 1/23/2015
Indication: Active immunization to prevent invasive meningococcal disease caused by N. meningitidis serogroup B in individuals 10 through 25 years of age
Category: Infectious Disease
Trade Name (Agent): Kalydeco (ivacaftor)
Sponsor: Vertex, Vertex Pharmaceuticals Inc.
Date of BT Designation Disclosure: 2013
Approval Date: 2/21/2014
Indication: Patients age 6 years and older with cystic fibrosis who have mutations in the CTFR gene
Category: Rare Inherited Disorders
Trade Name (Agent): Imbruvica (ibrutinib)
Sponsor:
Date of BT Designation Disclosure: 2/12/2013
Approval Date: 11/13/2013
Indication: Patients with mantle cell lymphoma (MCL)
Category: Cancer
Trade Name (Agent): Imbruvica (ibrutinib)
Sponsor:
Date of BT Designation Disclosure: 2/12/2013
Approval Date: 1/29/2015
Indication: Patients with Waldenstrom's macroglobulinemia (WM)
Category: Cancer
Trade Name (Agent): Zykadia (ceritinib)
Sponsor:
Date of BT Designation Disclosure: 3/15/2013
Approval Date: 4/29/2014
Indication: Patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib
Category: Cancer
Trade Name (Agent): Xalkori (crizotinib)
Sponsor:
Date of BT Designation Disclosure: 4/21/2015
Approval Date: 3/11/2016
Indication: Patients with non-small cell lung cancer whose tumors are ROS-1 positive
Category: Cancer
Trade Name (Agent): Rapamune (sirolimus)
Sponsor:
Date of BT Designation Disclosure: 2015
Approval Date: 5/28/2015
Indication: Patients with lymphangioleiomyomatosis (LAM)
Category: Other
Trade Name (Agent): Yescarta (axicabtagene ciloleucel)
Sponsor: Kite Pharma Inc.
Date of BT Designation Disclosure: 12/07/2015
Approval Date: 10/18/2017
Indication: Adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy
Category: Cancer
Trade Name (Agent): Kymriah (tisagenlecleucel)
Sponsor: Novartis Pharmaceuticals Corp.
Date of BT Designation Disclosure: 7/07/2014
Approval Date: 8/30/2017
Indication: Pediatric and young adult patients (age 3-25 years) with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse
Category: Cancer
Trade Name (Agent): Opdivo (nivolumab)
Sponsor: Bristol-Myers Squibb Company
Date of BT Designation Disclosure: 9/26/2014
Approval Date: 12/22/2014
Indication: Patients with unresectable or metastatic melanoma and disease progression following ipilimumab and, if BRAF V600+, a BRAF inhibitor
Category: Cancer
Trade Name (Agent): Opdivo (nivolumab)
Sponsor: Bristol-Myers Squibb Company
Date of BT Designation Disclosure: 9/02/2015
Approval Date: 10/09/2015
Indication: Patients with metastatic non-squamous non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy
Category: Cancer
Trade Name (Agent): Keytruda (pembrolizumab)
Sponsor: Merck Sharp & Dohme Corp
Date of BT Designation Disclosure: 4/24/2013
Approval Date: 12/18/2015
Indication: Patients with unresectable or metastatic melanoma
Category: Cancer
Trade Name (Agent): Ibrance (palbociclib)
Sponsor: Pfizer Inc.
Date of BT Designation Disclosure: 2016
Approval Date: 2/19/2016
Indication: Patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced metastatic breast cancer in combination with fulvestrant in women with disease progression following endocrine therapy
Category: Cancer
Trade Name (Agent): Gavreto (Pralsentinib)
Sponsor: Blueprint Medicines Corporation
Date of BT Designation Disclosure: 2/26/2019
Approval Date: 9/04/2020
Indication: Patients with RET fusion-positive NSCLC that has progressed on platinum-based chemotherapy
Category: Cancer
Trade Name (Agent): LN-145
Sponsor: Iovance Biotherapeutics, Inc.
Date of BT Designation Disclosure: 5/22/2019
Indication: Patients with recurrent, metastatic, or persistent cervical cancer who have progressed on or after chemotherapy
Category: Cancer
Trade Name (Agent): Zolgensma (onasemnogene abeparvovec-xioi)
Sponsor: AveXis
Date of BT Designation Disclosure: 7/20/2016
Approval Date: 5/24/2019
Indication: Spinal muscular atrophy (SMA) Type 1 in children less than two years old
Category: Other
Trade Name (Agent): enfortumab vedotin
Sponsor: Astellas Pharma US, Inc. and Seattle Genetics, Inc.
Date of BT Designation Disclosure: 3/26/2018
Indication: Patients with locally advanced or metastatic urothelial carcinoma
Category: Cancer
Trade Name (Agent): LJPC-0118
Sponsor: La Jolla Pharmaceutical Company
Date of BT Designation Disclosure: 4/24/2019
Indication: Patients with severe malaria
Category: Infectious Disease
Trade Name (Agent): elafibranor
Sponsor: Genfit
Date of BT Designation Disclosure: 4/19/2019
Indication: Patients with primary biliary cholangitis (PBC)
Category: Other
Trade Name (Agent): SER-109
Sponsor: Seres Therapeutics, Inc.
Date of BT Designation Disclosure: 4/29/2019
Indication: Recurrent C. difficile infection
Category: Rare Inherited Disorders
Trade Name (Agent): PB2452
Sponsor: PhaseBio Pharmaceuticals
Date of BT Designation Disclosure: 4/08/2019
Indication: Reversal agent for the antiplatelet drug ticagrelor
Category: Other
Trade Name (Agent): AXS-05
Sponsor: Axsome Therapeutics, Inc.
Date of BT Designation Disclosure: 3/27/2019
Indication: Major Depressive Disorder (MDD)
Category: Rare Inherited Disorders
Trade Name (Agent): Oliceridine (TRV130)
Sponsor: Trevena, Inc.
Date of BT Designation Disclosure: 2/22/2016
Indication: Moderate-to-Severe Acute Pain
Category: Other
Trade Name (Agent): Aliqopa (copanlisib)
Sponsor: Bayer
Date of BT Designation Disclosure: 5/30/2019
Indication: Adults patients with relapsed marginal zone lymphoma (MZL) who have received at least 2 prior therapies
Category: Cancer
Trade Name (Agent): Jakafi (Ruxolitinib)
Sponsor: Incyte
Date of BT Designation Disclosure: 6/23/2016
Approval Date: 5/28/2019
Indication: Patients aged 12 years and older with steroid-refractory acute graft versus-host disease (GVHD)
Category: Other
Trade Name (Agent): Givlaari (givosiran)
Sponsor: Alnylam Pharmaceuticals Inc.
Date of BT Designation Disclosure: 5/30/2017
Approval Date: 11/20/2019
Indication: Adult patients with acute hepatic porphyria (AHP)
Category: Rare Inherited Disorders
Trade Name (Agent): Trikafta (elexacaftor/ivacaftor/tezacaftor)
Sponsor: Vertex Pharmaceuticals Inc.
Date of BT Designation Disclosure: 5/01/2018
Approval Date: 10/21/2019
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
Trade Name (Agent): Zejula (niraparib)
Sponsor: Janssen Pharmaceutica
Date of BT Designation Disclosure: 10/03/2019
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
Trade Name (Agent): Cotellic (cobimetinib)
Sponsor: Memorial Sloan Kettering Cancer Center
Date of BT Designation Disclosure: 10/03/2019
Indication: Adult patients with histiocytic neoplasms who do not harbor a BRAF V600 mutation
Category: Rare Inherited Disorders
Trade Name (Agent): Tepotinib (Tepmetko)
Sponsor: Merck
Date of BT Designation Disclosure: 9/11/2019
Approval Date: 2/03/2021
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
Trade Name (Agent): Nirogacestat
Sponsor: SpringWorks Therapeutics, Inc.
Date of BT Designation Disclosure: 8/29/2019
Indication: Adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis
Category: Cancer
Trade Name (Agent): NKTR-214 (bempegaldesleukin) with Opdivo (nivolumab)
Sponsor: Nektar Therapeutics
Date of BT Designation Disclosure: 8/23/2019
Indication: Untreated melanoma patients with either metastatic disease or who are unable to undergo surgery
Category: Cancer
Trade Name (Agent): Peginterferon Lambda
Sponsor: Eiger Pharmaceuticals
Date of BT Designation Disclosure: 8/20/2019
Indication: Hepatitis delta virus (HDV) infection
Category: Infectious Disease
Trade Name (Agent): DCR-PHXC
Sponsor: Dicerna Pharmaceuticals
Date of BT Designation Disclosure: 7/15/2019
Indication: Primary hyperoxaluria type 1 (caused by a mutation in the AGXT gene)
Category: Other
Trade Name (Agent): Avexitide
Sponsor: Eiger BioPharmaceuticals Inc.
Date of BT Designation Disclosure: 6/17/2019
Indication: Post-bariatric hypoglycemia (PBH)
Category: Other
Trade Name (Agent): seladelpar
Sponsor: CymaBay Therapeutics
Date of BT Designation Disclosure: 2/20/2019
Indication: Patients with early stage primary biliary cholangitis (PBC), in combination with ursodeoxycholic acid (UDCA)
Category: Other
Trade Name (Agent): Braftovi (encorafenib) + Mektovi (binimetinib) + Erbitux (cetuximab)
Sponsor: Array BioPharma and Eli Lilly and Co.
Date of BT Designation Disclosure: 8/07/2018
Indication: Patients with BRAF V600E-mutation metastatic colorectal caner following 1 or 2 prior lines of treatment in the metastatic setting
Category: Cancer
Trade Name (Agent): Rezurock (belumosudil)
Sponsor: Kadmon Holdings, Inc.
Date of BT Designation Disclosure: 10/17/2018
Approval Date: 7/16/2021
Indication: Patients with chronic graft-versus-host disease (cGVHD) after failure of two or more lines of systemic therapy
Category: Other
Trade Name (Agent): Hemlibra (emicizumab-kxwh)
Sponsor: GENENTECH INC
Date of BT Designation Disclosure: 4/17/2018
Approval Date: 10/04/2018
Indication: Patients with hemophilia A without factor VIII inhibitors
Category: Rare Inherited Disorders
Trade Name (Agent): Rubraca (rucaparib)
Sponsor: Clovis Oncology
Date of BT Designation Disclosure: 10/03/2018
Indication: BRCA1/BRCA2-mutated, metastatic, castration-resistant prostate cancer
Category: Cancer
Trade Name (Agent): Luxturna (voretigene neparvovec-rzyl)
Sponsor: Spark Therapeutics Inc.
Date of BT Designation Disclosure: 9/24/2014
Approval Date: 12/19/2017
Indication: Patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy
Category: Rare Inherited Disorders
Trade Name (Agent): Prevnar 20 (PF-06482077)
Sponsor: Pfizer Inc.
Date of BT Designation Disclosure: 9/20/2018
Approval Date: 6/08/2021
Indication: Prevention of pneumonia and invasive disease caused by Streptococcus pneumoniae serotypes
Category: Infectious Disease
Trade Name (Agent): CA-008
Sponsor: Concentric Analgesics, Inc.
Date of BT Designation Disclosure: 9/11/2018
Indication: Patients needing long-lasting, non-opioid pain relief
Category: Other
Trade Name (Agent): Tezepelumab
Sponsor: AstraZeneca, AstraZeneca & Amgen Inc.
Date of BT Designation Disclosure: 9/07/2018
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
Trade Name (Agent): PF-06651600
Sponsor: Pfizer Inc.
Date of BT Designation Disclosure: 9/06/2018
Indication: Patients with alopecia areata
Category: Other
Trade Name (Agent): LOXO-292
Sponsor: Loxo Oncology, Inc.
Date of BT Designation Disclosure: 9/05/2018
Indication: Patients with metastatic RET-fusion-positive non-small cell lung cancer; patients with RET-mutant medullary thyroid cancer
Category: Cancer
Trade Name (Agent): Danyelza (naxitamab-gqgk)
Sponsor: Y-mAbs Therapeutics, Inc.
Date of BT Designation Disclosure: 8/23/2018
Approval Date: 11/25/2020
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
Trade Name (Agent): Poteligeo (mogamulizumab-kpkc)
Sponsor: Kyowa Kirin Inc.
Date of BT Designation Disclosure: 10/25/2017
Approval Date: 8/08/2018
Indication: Adult patients Sézary syndrome (SS) after at least one prior systemic therapy
Category: Cancer
Trade Name (Agent): LOXO-292
Sponsor: Loxo Oncology, Inc.
Date of BT Designation Disclosure: 10/16/2018
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
Trade Name (Agent): Myrcludex
Sponsor: MYR Pharma
Date of BT Designation Disclosure: 10/23/2018
Indication: Chronic hepatitis delta (HDV) infection
Category: Infectious Disease
Trade Name (Agent): Psilocybin therapy
Sponsor: COMPASS Pathways
Date of BT Designation Disclosure: 10/23/2018
Indication: Treatment-resistant Depression
Category: Other
Trade Name (Agent): AT-GAA (ATB200 + AT2221)
Sponsor: Amicus Therapeutics
Date of BT Designation Disclosure: 2/26/2019
Indication: Patients with late onset Pompe disease
Category: Rare Inherited Disorders
Trade Name (Agent): CP101
Sponsor: Finch Therapeutics Group, Inc.
Date of BT Designation Disclosure: 2/09/2019
Indication: Patients with recurrent Clostridium difficile infection
Category: Infectious Disease
Trade Name (Agent): UKONIQ (umbralisib)
Sponsor: TG Therapeutics
Date of BT Designation Disclosure: 1/28/2019
Approval Date: 2/05/2021
Indication: Adult patients with marginal zone lymphoma (MZL) who did not respond to at least one anti-CD20 therapy
Category: Cancer
Trade Name (Agent): Vaxneuvance (V114)
Sponsor: Merck & Co., Inc.
Date of BT Designation Disclosure: 1/28/2019
Approval Date: 7/16/2021
Indication: Prevention of invasive pneumococcal disease in adults 18 years and older caused by 15 serotypes.
Category: Infectious Disease
Trade Name (Agent): MEDI8897
Sponsor: AstraZeneca
Date of BT Designation Disclosure: 2/06/2019
Indication: Prevention of lower respiratory tract infection (LRTI) caused by respiratory syncytial virus (RSV)
Category: Other
Trade Name (Agent): lonafarnib
Sponsor: Eiger BioPharmaceuticals
Date of BT Designation Disclosure: 11/19/2018
Approval Date: 11/20/2020
Indication: Patients with Hutchinson-Gilford progeria syndrome (HGPS_
Category: Rare Inherited Disorders
Trade Name (Agent): Olipudase Alfa
Sponsor:
Date of BT Designation Disclosure: 11/29/2018
Indication: Niemann-Pick disease Type B (NPB)
Category: Rare Inherited Disorders
Trade Name (Agent): Andexxa (andexanet alfa)
Sponsor:
Date of BT Designation Disclosure: 11/25/2013
Approval Date: 5/03/2018
Indication: patients treated with rivaroxaban and apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding
Category: Cardiovascular
Trade Name (Agent): Xolair (omalizumab)
Sponsor:
Date of BT Designation Disclosure: 8/13/2018
Approval Date: 9/28/2018
Indication: Prevention of severe allergic reactions following accidental exposure to one or more foods in people with allergies
Category: Other
Trade Name (Agent): NGX-101
Sponsor:
Date of BT Designation Disclosure: 11/13/2018
Indication: Severe Bipolar Depression with Acute Suicidal Ideation & Behavior (ASIB) after initial stabilization with ketamine or other effective therapy
Category: Other
Trade Name (Agent): Promacta (eltrombopag)
Sponsor:
Date of BT Designation Disclosure: 11/16/2018
Indication: Low platelet counts in people exposed to radiation
Category: Other
Trade Name (Agent): Breyanzi (Lisocabtagene maraleucel)
Sponsor:
Date of BT Designation Disclosure: 12/20/2016
Approval Date: 2/05/2021
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
Trade Name (Agent): Rozlytrek (entrectinib)
Sponsor: GENENTECH INC
Date of BT Designation Disclosure: 5/15/2017
Approval Date: 8/15/2019
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
Trade Name (Agent): Turalio (pexidartinib)
Sponsor: Daiichi Sankyo
Date of BT Designation Disclosure: 10/30/2015
Approval Date: 8/02/2019
Indication: Adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery
Category: Cancer
Trade Name (Agent): Polivy (polatuzumab vedotin-piiq)
Sponsor: GENENTECH INC
Date of BT Designation Disclosure: 12/10/2017
Approval Date: 6/10/2019
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
Trade Name (Agent): Emgality (galcanezumab)
Sponsor: Eli Lilly & Company
Date of BT Designation Disclosure: 11/15/2018
Approval Date: 6/04/2019
Indication: Episodic cluster headache in adults
Category: Rare Inherited Disorders
Trade Name (Agent): Venclexta (venetoclax)
Sponsor: AbbVie Inc.
Date of BT Designation Disclosure: 3/15/2019
Approval Date: 5/15/2019
Indication: In combination with obinutuzumab for treatment of previously untreated patients with chronic lymphocytic leukemia (1L CLL) or small lymphocytic lymphoma (SLL)
Category: Cancer
Trade Name (Agent): Bavencio (avelumab)
Sponsor: EMD Serono Inc.
Date of BT Designation Disclosure: 12/22/2017
Approval Date: 5/14/2019
Indication: In combination with Inlyta (axitinib) for first-line treatment of patients with advanced renal cell carcinoma (RCC)
Category: Cancer
Trade Name (Agent): Kadcyla (ado-trastuzumab emtansine)
Sponsor: GENENTECH INC
Date of BT Designation Disclosure: 2/05/2019
Approval Date: 5/03/2019
Indication: Adjuvant treatment of patients with HER2-positive early breast cancer who have residual disease after neoadjuvant taxane and trastuzumab-based treatment
Category: Cancer
Trade Name (Agent): Vyndaquel (tafamidis meglumine)
Sponsor: Foldrx Pharmaceuticals, Inc. Sub. Pfizer, Inc.
Date of BT Designation Disclosure: 5/24/2018
Approval Date: 5/03/2019
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
Trade Name (Agent): Keytruda (pembrolizumab)
Sponsor: Merck Sharpe & Dohme Corp.
Date of BT Designation Disclosure: 12/10/2017
Approval Date: 4/19/2019
Indication: In combination with axitinib for the first-line treatment of patients with advanced renal cell carcinoma (RCC)
Category: Cancer
Trade Name (Agent): Balversa (erdafitinib)
Sponsor: Janssen Biotech Inc.
Date of BT Designation Disclosure: 3/15/2018
Approval Date: 4/12/2019
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
Trade Name (Agent): Ibrance (palbociclib)
Sponsor: Pfizer Inc.
Date of BT Designation Disclosure: 2017
Approval Date: 4/04/2019
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
Trade Name (Agent): Keytruda (pembrolizumab)
Sponsor: Merck Sharp & Dohme Corp
Date of BT Designation Disclosure: 8/01/2018
Approval Date: 9/17/2019
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
Trade Name (Agent): Lenvima (lenvatinib)
Sponsor: Eisai Co.
Date of BT Designation Disclosure: 8/01/2018
Approval Date: 9/17/2019
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
Trade Name (Agent): Oxbryta (voxelotor)
Sponsor: Global Blood Therapeutics, Inc.
Date of BT Designation Disclosure: 1/09/2018
Approval Date: 11/25/2019
Indication: Sickle cell disease (SCD) in patients 12 years and older
Category: Rare Inherited Disorders
Trade Name (Agent): Enhertu (fam-trastuzumab deruxtecan-nxki)
Sponsor: Daiichi Sankyo Co.
Date of BT Designation Disclosure: 5/08/2019
Approval Date: 12/20/2019
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
Trade Name (Agent): B38M (JNJ‐4528)
Sponsor: Legend Biotech + Janssen Global Services
Date of BT Designation Disclosure: 12/06/2019
Indication: Patients with previously treated multiple myeloma
Category: Cancer
Trade Name (Agent): Cosela (trilaciclib)
Sponsor: Cosela to G1 Therapeutics, Inc.
Date of BT Designation Disclosure: 8/07/2019
Approval Date: 2/12/2021
Indication: Reduce the frequency of chemotherapy-induced bone marrow suppression in adults receiving certain types of chemotherapy for extensive-stage (when the cancer has spread beyond the lungs) small cell lung cancer.
Category: Cancer
Trade Name (Agent): Tibosovo (ivosidenib)
Sponsor: Servier Pharmaceuticals
Date of BT Designation Disclosure: 3/27/2019
Approval Date: 5/25/2022
Indication: In combination with azacitidine or as monotherapy for the treatment of newly diagnosed acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy.
Category: Cancer
Trade Name (Agent): omidubicel
Sponsor: Gamida Cell Ltd.
Date of BT Designation Disclosure: 10/11/2016
Indication: Patients with high-risk blood cancers who do not have a suitable matched donor
Category: Cancer
Trade Name (Agent): Padcev (enfortumab vedotin-ejfv)
Sponsor: Astellas Pharma US, Inc.
Date of BT Designation Disclosure: 3/26/2018
Approval Date: 12/18/2019
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
Trade Name (Agent): Calquence (acalabrutinib)
Sponsor: AstraZeneca
Date of BT Designation Disclosure: 8/14/2019
Approval Date: 11/21/2019
Indication: Adult patients with relapsed or refractory chronic lymphocytic leukemia
Category: Cancer
Trade Name (Agent): Calquence (acalabrutinib)
Sponsor: AstraZeneca
Date of BT Designation Disclosure: 8/14/2019
Approval Date: 11/21/2019
Indication: Adult patients with untreated chronic lymphocytic leukemia (CLL)
Category: Cancer
Trade Name (Agent): Adakveo (crizanlizumab-tmca)
Sponsor: Novartis Pharmaceuticals Corporation
Date of BT Designation Disclosure: 1/08/2019
Approval Date: 11/15/2019
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
Trade Name (Agent): Brukinsa (zanubrutinib)
Sponsor: BeiGene USA, Inc.
Date of BT Designation Disclosure: 1/16/2019
Approval Date: 11/14/2019
Indication: Patients with mantle cell lymphoma (MCL) who have received at least one prior therapy
Category: Cancer
Trade Name (Agent): Zulresso (Brexanolone)
Sponsor: Sage Therapeutics Inc.
Date of BT Designation Disclosure: 9/06/2016
Approval Date: 3/19/2019
Indication: Postpartum depression (PPD)
Category: Other
Trade Name (Agent): Venclexta (venetoclax)
Sponsor: AbbVie Inc.
Date of BT Designation Disclosure: 10/25/2017
Approval Date: 11/21/2018
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
Trade Name (Agent): Oxervate (cenegermin-bkbj ophthalmic solution)
Sponsor: Dompe Farmaceutici SpA
Date of BT Designation Disclosure: 11/30/2017
Approval Date: 8/22/2018
Indication: Patients with neurotrophic keratitis (NK)
Category: Other
Trade Name (Agent): Onpattro (patisiran)
Sponsor: Alnylam Pharmaceuticals Inc.
Date of BT Designation Disclosure: 2/02/2018
Approval Date: 8/10/2018
Indication: Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
Category: Rare Inherited Disorders
Trade Name (Agent): Orkambi (lumacaftor/ivacaftor)
Sponsor: Vertex Pharmaceuticals Inc.
Date of BT Designation Disclosure: 3/12/2018
Approval Date: 8/07/2018
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
