Trade Name (Agent)
Sponsor
Date of BT Designation Disclosure
Approval Date
Indication
Category
Trade Name (Agent): Kalydeco (ivacaftor)
Sponsor: Vertex Pharmaceuticals Inc.
Date of BT Designation Disclosure: 2014
Approval Date: 12/29/2014
Indication: Patients age 6 years and older with cystic fibrosis who have the R117H mutation in the CF transmembrane conductance regulator (CTFR gene)
Category: Rare Inherited Disorders
Trade Name (Agent): Ibrance (palbociclib)
Sponsor: Pfizer Inc.
Date of BT Designation Disclosure: 4/10/2013
Approval Date: 2/03/2015
Indication: Postmenopausal women with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced metastatic breast cancer as initial endocrine-based therapy for their metastatic disease
Category: Cancer
Trade Name (Agent): Palforzia (Arachis hypogaea)
Sponsor: Aimmune
Date of BT Designation Disclosure: 6/18/2015
Approval Date: 1/31/2020
Indication: An oral immunotherapy indicated for the mitigation of allergic reactions, including anaphylaxis, that may occur with accidental exposure to peanut.
Category: Other
Trade Name (Agent): ARCALYST (rilonacept)
Sponsor: Kiniksa
Date of BT Designation Disclosure: 11/20/2019
Approval Date: 3/18/2021
Indication: Treatment of recurrent pericarditis
Category: Cardiovascular
Trade Name (Agent): NRX-101
Sponsor: NeuroRx
Date of BT Designation Disclosure: 11/13/2018
Indication: Treatment of Severe Bipolar Depression with Acute Suicidal Ideation & Behavior (ASIB)
Category: Other
Trade Name (Agent): Viaskin® Peanut
Sponsor: DBV Therapies
Date of BT Designation Disclosure: 4/09/2015
Indication: Treatment for patients suffering from life threatening food allergies
Category: Other
Trade Name (Agent): Omidubicel (formerly known as NiCord®)
Sponsor: Gamida Cell (Emmes)
Date of BT Designation Disclosure: 10/11/2016
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
Trade Name (Agent): Brensocatib
Sponsor: Insmed Incorporated
Date of BT Designation Disclosure: 6/18/2020
Indication: Treatment for bronchiectasis
Category: Rare Inherited Disorders
Trade Name (Agent): AXS-05
Sponsor: Axsome Therapeutics
Date of BT Designation Disclosure: 6/26/2020
Indication: Treatment of Alzheimer’s disease agitation, major depressive disorder, and other central nervous system (CNS) disorders
Category: Other
Trade Name (Agent): TLX250-CDX
Sponsor: Telix Pharmaceuticals
Date of BT Designation Disclosure: 7/01/2020
Indication: Renal cancer imaging product
Category: Cancer
Trade Name (Agent): ER-004
Sponsor: Esperare
Date of BT Designation Disclosure: 7/06/2020
Indication: Investigational Prenatal Treatment ER-004 In X-Linked Hypohidrotic Ectodermal Dysplasia
Category: Rare Inherited Disorders
Trade Name (Agent): Padcev
Sponsor: Astellas Pharma
Date of BT Designation Disclosure: 2/20/2020
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
Trade Name (Agent): CD20xCD3
Sponsor: F. Hoffmann-La Roche AG
Date of BT Designation Disclosure: 7/14/2020
Indication: Treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma who have received at least two prior systemic therapies
Category: Cancer
Trade Name (Agent): Olorofim (F901318)
Sponsor: F2G The Rare Fungal Disease Company
Date of BT Designation Disclosure: 11/13/2019
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
Trade Name (Agent): APR-246
Sponsor: Aprea Therapeutics
Date of BT Designation Disclosure: 1/30/2020
Indication: Treatment of patients with myelodysplastic syndromes with a susceptible TP53 mutation
Category: Cancer
Trade Name (Agent): JNJ-6372
Sponsor: Janssen Biotech, Inc.
Date of BT Designation Disclosure: 3/10/2020
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
Trade Name (Agent): AXS-05
Sponsor: Axsome Therapeutics, Inc.
Date of BT Designation Disclosure: 3/15/2019
Indication: Patients with major depressive disorder that is resistant to current treatments
Category: Other
Trade Name (Agent): ABECMA (idecabtagene vicleucel)
Sponsor: Bristol-Myers Squibb Company
Date of BT Designation Disclosure: 11/15/2017
Approval Date: 3/26/2021
Indication: Adult patients with relapsed or refractory multiple myeloma
Category: Cancer
Trade Name (Agent): Sotatercept
Sponsor: Acceleron Pharma
Date of BT Designation Disclosure: 4/08/2020
Indication: Patients with pulmonary arterial hypertension
Category: Cardiovascular
Trade Name (Agent): Esbriet (pirfenidone)
Sponsor: Hoffman-La Roche Inc.
Date of BT Designation Disclosure: 3/03/2020
Indication: Patients with unclassifiable interstitial lung disease
Category: Other
Trade Name (Agent): Baricitinib
Sponsor: Eli Lilly and Company + Incyte Corporation
Date of BT Designation Disclosure: 3/16/2020
Indication: Patients with alopecia areata
Category: Other
Trade Name (Agent): Psilocybin formulation (Comp 360)
Sponsor: Compass Pathways
Date of BT Designation Disclosure: 10/23/2018
Indication: Treatment-resistant depression
Category: Other
Trade Name (Agent): Neuromodulation therapy (NTX100)
Sponsor: Noctrix Health
Date of BT Designation Disclosure: 6/02/2020
Indication: Treatment of adults with primary moderate-severe Restless Legs Syndrome (RLS) who are refractory to medications
Category: Other
Trade Name (Agent): HSV Pritelivir (AIC316)
Sponsor: Anti-infective Cures GmbH
Date of BT Designation Disclosure: 6/05/2020
Indication: Treatment of acyclovir-resistant mucocutaneous herpes simplex virus (HSV) infections in immunocompromised adults
Category: Other
Trade Name (Agent): Brensocatib (INS1007)
Sponsor: Insmed Incorporated
Date of BT Designation Disclosure: 6/08/2020
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
Trade Name (Agent): Zynteglo (betibeglogene autotemcel (beti-cel; formerly LentiGlobin™))
Sponsor: BlueBird
Date of BT Designation Disclosure: 2/02/2015
Indication: Beta-thalassemia
Category: Rare Inherited Disorders
Trade Name (Agent): Ofev (nintedanib)
Sponsor: Boehringer Ingelheim Pharmaceuticals
Date of BT Designation Disclosure: 10/10/2019
Approval Date: 3/09/2020
Indication: Chronic fibrosing interstitial lung diseases (ILDs) with a progressive phenotype
Category: Rare Inherited Disorders
Trade Name (Agent): CTP-543
Sponsor: Concert Pharmaceuticals
Date of BT Designation Disclosure: 7/08/2020
Indication: Treatment of Alopecia Areata
Category: Rare Inherited Disorders
Trade Name (Agent): Sacituzumab Govitecan-hziy
Sponsor: Immunomedics INC
Date of BT Designation Disclosure: 2/05/2016
Approval Date: 4/22/2020
Indication: Treatment of adult patients with metastatic triple-negative breast cancer (mTNBC) who have received at least two prior therapies for metastatic disease
Category: Cancer
Trade Name (Agent): TABRECTA (capmatinib)
Sponsor: Novartis Pharmaceuticals Corporation
Date of BT Designation Disclosure: 9/06/2019
Approval Date: 5/06/2020
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
Trade Name (Agent): Retevmo (selpercatinib)
Sponsor: Loxo Oncology, Inc.
Date of BT Designation Disclosure: 10/15/2018
Approval Date: 5/08/2020
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
Trade Name (Agent): Yervoy (ipilimumab)
Sponsor: Bristol-Myers Squibb Company
Date of BT Designation Disclosure: 3/10/2020
Indication: In combination with nivolumab, for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib
Category: Cancer
Trade Name (Agent): Opdivo (nivolumab)
Sponsor: Bristol-Myers Squibb Company
Date of BT Designation Disclosure: 2020
Approval Date: 3/10/2020
Indication: In combination with ipilimumab, for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib
Category: Cancer
Trade Name (Agent): Jelmyto (mitomycin)
Sponsor: UroGen Pharma
Date of BT Designation Disclosure: 10/31/2018
Approval Date: 4/15/2020
Indication: Treatment of adult patients with lowgrade upper tract urothelial cancer
Category: Cancer
Trade Name (Agent): Pemazyre (pemigatinib)
Sponsor: Incyte Corporation
Date of BT Designation Disclosure: 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
Trade Name (Agent): Qinlock (Ripretinib)
Sponsor: DECIPHERA PHARMACEUTICALS LLC
Date of BT Designation Disclosure: 2020
Approval Date: 5/15/2020
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
Trade Name (Agent): Artesunate
Sponsor: AMIVAS LLC
Date of BT Designation Disclosure: 2020
Approval Date: 5/26/2020
Indication: Initial treatment of severe malaria in adult and pediatric patients
Category: Other
Trade Name (Agent): Enspryng (satralizumab-mwge)
Sponsor: Genentech, Inc.
Date of BT Designation Disclosure: 12/20/2018
Approval Date: 8/14/2020
Indication: Subcutaneous treatment for adults living with anti-aquaporin-4 (AQP4) antibody positive neuromyelitis optica spectrum disorder (NMOSD).
Category: Rare Inherited Disorders
Trade Name (Agent): TUKYSA (tucatinib)
Sponsor: SEATTLE GENETICS INC
Date of BT Designation Disclosure: 2019
Approval Date: 4/17/2020
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
Trade Name (Agent): Koselugo (Selumetinib)
Sponsor: AstraZeneca Plc and Merck & Co., Inc.
Date of BT Designation Disclosure: 4/01/2019
Approval Date: 4/10/2020
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
Trade Name (Agent): Rukobia (fostemsavir)
Sponsor: ViiV Healthcare
Date of BT Designation Disclosure: 7/21/2015
Approval Date: 7/02/2020
Indication: Antiretroviral medication for adults living with HIV who have tried multiple HIV medications and whose HIV infection cannot be successfully treated with other therapies because of resistance, intolerance or safety considerations.
Category: Infectious Disease
Trade Name (Agent): Mavacamten (MYK-461)
Sponsor: MyoKardia
Date of BT Designation Disclosure: 7/23/2020
Indication: Treatment of symptomatic, obstructive hypertrophic cardiomyopathy (HCM).
Category: Rare Inherited Disorders
Trade Name (Agent): MK-6482
Sponsor: Merck Sharpe & Dohme Corp.
Date of BT Designation Disclosure: 7/29/2020
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
Trade Name (Agent): Tagrisso (osimertinib)
Sponsor: AstraZeneca
Date of BT Designation Disclosure: 7/30/2020
Approval Date: 12/18/2020
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
Trade Name (Agent): Bavencio (avelumab)
Sponsor: Merck Sharpe & Dohme Corp.
Date of BT Designation Disclosure: 4/09/2020
Approval Date: 7/01/2020
Indication: Patients with locally advanced or metastatic urothelial carcinoma that has not progressed with first-line platinum-containing chemotherapy
Category: Cancer
Trade Name (Agent): Pevonedistat
Sponsor: Takeda Pharmaceutical Company
Date of BT Designation Disclosure: 7/30/2020
Indication: Treatment of patients with higher-risk myelodysplastic syndromes (HR-MDS)
Category: Cancer
Trade Name (Agent): AXS-12
Sponsor: Axsome Therapeutics
Date of BT Designation Disclosure: 8/05/2020
Indication: Treatment of cataplexy in patients with narcolepsy.
Category: Other
Trade Name (Agent): TEPEZZA (TEPROTUMUMABTRBW)
Sponsor: HORIZON THERAPEUTICS IRELAND DAC
Date of BT Designation Disclosure: 2020
Approval Date: 1/21/2020
Indication: Treatment of thyroid eye disease
Category: Other
Trade Name (Agent): Ayvakit (avapritinib)
Sponsor: Blueprint Medicines Corporation
Date of BT Designation Disclosure: 6/01/2017
Approval Date: 1/09/2020
Indication: Patients with unresectable or metastatic GIST harboring the PDGFRα D842V mutation
Category: Cancer
Trade Name (Agent): Tecentriq (atezolizumab)
Sponsor: Genentech Inc.
Date of BT Designation Disclosure: 7/18/2018
Approval Date: 5/29/2020
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
Trade Name (Agent): Uplinza (inebilizumab-cdon)
Sponsor: Viela Bio
Date of BT Designation Disclosure: 4/18/2019
Approval Date: 6/11/2020
Indication: Patients with neuromyelitis optica spectrum disorder (NMOSD)
Category: Rare Inherited Disorders
