Trade Name (Agent): INO-3107
Sponsor: INOVIO Pharmaceuticals Inc.
Date of BT Designation Disclosure: 9/7/2023
Indication: Treatment for patients with Recurrent Respiratory Papillomatosis (RRP)
Category: Cancer
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 March 31, 2023, the FDA has approved 278* breakthrough therapy designated products and lists that there have been 1342 total requests for the designation with 519** designations granted.
Trade Name (Agent)
Sponsor
Date of BT Designation Disclosure
Approval Date
Indication
Category
Trade Name (Agent): NS-089/NCNP-02
Sponsor: NS Pharma
Date of BT Designation Disclosure: 7/27/2023
Indication: Investigational candidate for patients with Duchenne muscular dystrophy amenable to exon 44 skipping therapy.
Category: Other
Trade Name (Agent): RBT-1
Sponsor: Renibus Therapeutics
Date of BT Designation Disclosure: 7/11/2023
Indication: Reduction in risk of post-operative complications in patients undergoing cardiothoracic surgery
Category: Other
Trade Name (Agent): Zenocutuzumab (Zeno)
Sponsor: Merus N.V.
Date of BT Designation Disclosure: 6/29/2023
Indication: Treatment of patients with advanced unresectable or metastatic NRG1 fusion (NRG1+) pancreatic cancer following progression with prior systemic therapy or who have no satisfactory alternative treatment options.
Category: Cancer
Trade Name (Agent): PRGN-2012 AdenoVerse™
Sponsor: Precigen
Date of BT Designation Disclosure: 6/20/2023
Indication: Treatment of recurrent respiratory papillomatosis (RRP).
Category: Other
Trade Name (Agent): Resiniferatoxin (RTX)
Sponsor: Grünenthal
Date of BT Designation Disclosure: 5/22/2023
Indication: Treatment for pain associated with osteoarthritis (OA) of the knee.
Category: Other
Trade Name (Agent): Spesolimab
Sponsor:
Date of BT Designation Disclosure: 5/2/2023
Indication: Investigational treatment for the prevention of flares in adults with generalized pustular psoriasis (GPP).
Category: Other
Trade Name (Agent): SAB-176
Sponsor: SAB Biotherapeutics
Date of BT Designation Disclosure: 4/18/2023
Indication: Therapeutic for post-exposure prophylaxis for Type A and Type B influenza illness in high-risk patients, including those who have anti-viral resistant strains.
Category: Infectious Disease
Trade Name (Agent): Resmetirom
Sponsor: Madrigal Pharmaceuticals
Date of BT Designation Disclosure: 4/18/2023
Indication: Treatment of patients with NASH with liver fibrosis.
Category: Other
Trade Name (Agent): mRNA-4157/V940 in combination with KEYTRUDA
Sponsor: Merck, Moderna
Date of BT Designation Disclosure: 2/22/2023
Indication: Adjuvant treatment of patients with high-risk melanoma following complete resection.
Category: Cancer
Trade Name (Agent): Pimicotinib (ABSK021)
Sponsor: Abbisko Therapeutics
Date of BT Designation Disclosure: 1/30/2023
Indication: 1/30/2023
Category: Other
Trade Name (Agent): mRNA-1345
Sponsor: Moderna
Date of BT Designation Disclosure: 1/30/2023
Indication: Vaccine for respiratory syncytial virus (RSV)
Category: Infectious Disease
Trade Name (Agent): VAX-24
Sponsor: Vaxcyte
Date of BT Designation Disclosure: 1/5/2023
Indication: Investigational 24-valent pneumococcal conjugate vaccine (PCV) candidate for the prevention of invasive pneumococcal disease (IPD)
Category: Infectious Disease
Trade Name (Agent): Pegsitacianine
Sponsor: OncoNano Medicine
Date of BT Designation Disclosure: 1/4/2023
Indication: Adjunct for the visualization of metastatic disease in the peritoneal cavity in patients undergoing cytoreductive surgery.
Category: Cancer
Trade Name (Agent): Adagrasib (KRAZATI™)
Sponsor: Mirata Therapeutics Inc.
Date of BT Designation Disclosure: 12/21/2022
Indication: Treatment in combination with cetuximab in patients with KRASG12C-mutated advanced colorectal cancer (CRC) whose cancer has progressed following prior treatment with chemotherapy and an anti-VEGF therapy.
Category: Cancer
Trade Name (Agent): Efruxifermin (EFX)
Sponsor: Akero Therapeutics
Date of BT Designation Disclosure: 12/7/2022
Indication: Treatment of nonalcoholic steatohepatitis (NASH).
Category: Other
Trade Name (Agent): Revumenib
Sponsor: Syndax
Date of BT Designation Disclosure: 12/4/2022
Indication: Treatment of adult and pediatric patients with relapsed or refractory (R/R) acute leukemia harboring a KMT2A rearrangement (KMT2Ar).
Category: Cancer
Trade Name (Agent): Zimura (avacincaptad pegol)
Sponsor: IVERIC bio
Date of BT Designation Disclosure: 11/17/2022
Indication: Treatment of geographic atrophy (GA) secondary to Age-Related Macular Degeneration (AMD).
Category: Other
Trade Name (Agent): Elranatamab
Sponsor: Pfizer Inc.
Date of BT Designation Disclosure: 11/3/2022
Approval Date: 8/14/2023
Indication: Treatment of people with relapsed or refractory multiple myeloma (RRMM).
Category: Cancer
Trade Name (Agent): Setmelanotide
Sponsor: Rhythm Pharmaceutical, Inc.
Date of BT Designation Disclosure: 11/1/2022
Indication: Treatment of hypothalamic obesity.
Category: Other
Trade Name (Agent): GBS6 (PF-06760805)
Sponsor: Pfizer Inc.
Date of BT Designation Disclosure: 9/7/2022
Indication: Vaccine for the prevention of invasive GBS disease due to the vaccine serotypes in newborns and young infants by active immunization of their mothers during pregnancy.
Category: Infectious Disease
Trade Name (Agent): Taletrectinib
Sponsor: AnHeart Therapeutics
Date of BT Designation Disclosure: 8/3/2022
Indication: Treatment of adult patients with advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC) who are ROS1 tyrosine kinase inhibitor (TKI) treatment naïve or previously treated with crizotinib.
Category: Cancer
Trade Name (Agent): Imcivree (setmelanotide)
Sponsor: Rhythm Pharmaceutical, Inc.
Date of BT Designation Disclosure: 1/7/2016
Approval Date: 6/16/2022
Indication: For chronic weight management in adult and pediatric patients 6 years of age and older with Bardet-Biedl Syndrome (BBS).
Category: Other
Trade Name (Agent): Gedatolisib
Sponsor: Celcuity Inc.
Date of BT Designation Disclosure: 7/18/2022
Indication: Treatment of patients with HR+/HER2- metastatic breast cancer whose disease progressed during treatment with a CDK4/6 therapy and a non-steroidal aromatase inhibitor.
Category: Cancer
Trade Name (Agent): Dupixent (dupilumab)
Sponsor: Regeneron Pharmaceuticals Inc.
Date of BT Designation Disclosure: 10/1/2016
Approval Date: 6/7/2022
Indication: Treatment of adult and pediatric patients ages 6 months and older with moderateto-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable.
Category: Other
Trade Name (Agent): VIJOICE (alpelisib)
Sponsor: Novartis Pharmaceuticals Corp.
Date of BT Designation Disclosure: 11/13/2019
Approval Date: 4/5/2022
Indication: Treatment of adult and pediatric patients 2 years of age and older with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS) who require systemic therapy.
Category: Cancer
Trade Name (Agent): Apretude (Cabotegravir)
Sponsor: ViiV Healthcare
Date of BT Designation Disclosure: 11/17/2020
Approval Date: 12/20/2021
Indication: HIV-1 pre-exposure prophylaxis (PrEP) to reduce the risk of sexually acquired HIV-1 infection in at-risk adults and pediatric patients 12 to less than 18 years of age weighing at least 35 kg.
Category: Infectious Disease
Trade Name (Agent): Oxbryta (voxelotor)
Sponsor: Global Blood Therapeutics, Inc.
Date of BT Designation Disclosure: 1/3/2018
Approval Date: 12/17/2021
Indication: Treatment of sickle cell disease in adults and pediatric patients 4 years of age and older.
Category: Rare Inherited Disorders
Trade Name (Agent): Oxbryta (voxelotor)
Sponsor: Global Blood Therapeutics, Inc.
Date of BT Designation Disclosure: 1/3/2018
Approval Date: 12/17/2021
Indication: Treatment of sickle cell disease in pediatric patients 4 years of age and older.
Category: Rare Inherited Disorders
Trade Name (Agent): Orencia (abatacept)
Sponsor: Bristol-Myers Squibb Co.
Date of BT Designation Disclosure: 12/4/2019
Approval Date: 12/15/2021
Indication: Prevention of moderate to severe acute graft-versus-host disease (GvHD) in hematopoietic stem cell transplants from unrelated donors.
Category: Other
Trade Name (Agent): Fyarro (ABI-009)
Sponsor: Aadi Bioscience Inc.
Date of BT Designation Disclosure: 1/3/2019
Approval Date: 11/22/2021
Indication: Treatment of adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa).
Category: Cancer
Trade Name (Agent): CARVYKTI (ciltacabtagene autoleucel)
Sponsor: Janssen Pharmaceuticals Inc.
Date of BT Designation Disclosure: 2022
Approval Date: 2/28/2022
Indication: Treatment of adult patients with relapsed or refractory multiple myeloma, who previously received a proteasome inhibitor (PI), an immunomodulatory agent (IMiD) and an anti-CD38 antibody.
Category: Cancer
Trade Name (Agent): Dupixent (dupilumab)
Sponsor: Regeneron Pharmaceuticals Inc.
Date of BT Designation Disclosure: 9/14/2020
Approval Date: 5/20/2022
Indication: Treatment of patients 12 years and older with eosinophilic esophagitis (EoE).
Category: Other
Trade Name (Agent): Tecartus (brexucabtagene autoleuce)
Sponsor: Kite Pharma Inc.
Date of BT Designation Disclosure: 2016
Approval Date: 7/24/2020
Indication: Chimeric antigen receptor or CAR T cell therapy for the treatment of adult patients with relapsed or refractory mantle cell lymphoma or MCL.
Category: Cancer
Trade Name (Agent): Talquetamab
Sponsor: Janssen Pharmaceuticals Inc.
Date of BT Designation Disclosure: 6/29/2022
Approval Date: 8/9/2023
Indication: Treatment of adult patients with relapsed or refractory multiple myeloma, who have previously received at least 4 prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody.
Category: Cancer
Trade Name (Agent): Inaxaplin (VX-147)
Sponsor: Vertex Pharmaceuticals, Inc.
Date of BT Designation Disclosure: 6/8/2022
Indication: Treatment for APOL1-mediated focal segmental glomerulosclerosis (FSGS).
Category: Other
Trade Name (Agent): Efanesoctocog alfa
Sponsor: Sanofi, Sobi
Date of BT Designation Disclosure: 6/1/2022
Indication: Treatment of people with hemophilia A.
Category: Other
Trade Name (Agent): TMB-001
Sponsor: Timber Pharmaceuticals, Inc.
Date of BT Designation Disclosure: 5/31/2022
Indication: Treatment of congenital ichthyosis (CI).
Category: Other
Trade Name (Agent): Repotrectinib
Sponsor: Turning Point Therapeutics Inc.
Date of BT Designation Disclosure: 5/10/2022
Indication: Treatment of patients with ROS1-positive metastatic non-small cell lung cancer (NSCLC) who have been previously treated with one ROS1 tyrosine kinase inhibitor and who have not received prior platinum-based chemotherapy.
Category: Cancer
Trade Name (Agent): ENHERTU (fam-trastuzumab deruxtecan-nxki)
Sponsor: AstraZeneca, Daiichi Sankyo
Date of BT Designation Disclosure: 4/26/2022
Approval Date: 5/4/2022
Indication: Treatment of adult patients with unresectable or metastatic HER2 low (IHC 1+ or IHC 2+/ISH-negative) breast cancer who have received a prior systemic therapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy.
Category: Cancer
Trade Name (Agent): CMS I-neb
Sponsor: Zambon
Date of BT Designation Disclosure: 4/21/2022
Indication: Treatment for the reduction in the incidence of pulmonary exacerbations in adult patients with non-cystic fibrosis bronchiectasis (NCFB) colonized with P. aeruginosa.
Category: Other
Trade Name (Agent): V116
Sponsor: Merck & Co., Inc.
Date of BT Designation Disclosure: 4/14/2022
Indication: Vaccine for the prevention of invasive pneumococcal disease (IPD) and pneumococcal pneumonia caused by Streptococcus pneumoniae serotypes 3, 6A/C, 7F, 8, 9N, 10A, 11A, 12F, 15A, 15B/C, 16F, 17F, 19A, 20, 22F, 23A, 23B, 24F, 31, 33F, 35B in adults 18 years of age and older.
Category: Infectious Disease
Trade Name (Agent): EDSIVO™ (celiprolol)
Sponsor: Acer Therapeutics Inc.
Date of BT Designation Disclosure: 4/4/2022
Indication: Treatment of patients with COL3A1-positive vascular Ehlers-Danlos syndrome (vEDS).
Category: Other
Trade Name (Agent): PF-06928316 (RSVpreF)
Sponsor: Pfizer Inc.
Date of BT Designation Disclosure: 3/24/2022
Indication: Vaccine for the prevention of lower respiratory tract disease caused by RSV in individuals 60 years of age or older.
Category: Infectious Disease
Trade Name (Agent): PF-06928316 (RSVpreF)
Sponsor: Pfizer Inc.
Date of BT Designation Disclosure: 03/02/2022
Indication: Vaccine for the prevention of RSV-associated lower respiratory tract illness in infants from birth up to six months of age by active immunization of pregnant women.
Category: Infectious Disease
Trade Name (Agent): BI 1015550
Sponsor: Boehringer Ingelheim
Date of BT Designation Disclosure: 02/24/2022
Indication: Treatment of idiopathic pulmonary fibrosis (IPF).
Category: Other
Trade Name (Agent): MVA-BN RSV
Sponsor: Bavarian Nordis A/S
Date of BT Designation Disclosure: 2/14/2022
Indication: Active immunization for prevention of lower respiratory tract disease caused by respiratory syncytial virus (RSV) in adults aged 60 years or older.
Category: Infectious Disease
Trade Name (Agent): Farxiga® (dapagliflozin)
Sponsor: AstraZeneca
Date of BT Designation Disclosure: 1/29/2022
Indication: Treatment of patients with chronic kidney disease (CKD) whether or not they have type 2 diabetes.
Category: Other
Trade Name (Agent): DZD9008 (Sunvozertinib)
Sponsor: Dizal Pharmaceutical Co., Ltd.
Date of BT Designation Disclosure: 1/27/2022
Indication: Treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon20 insertion mutations whose disease has progressed on or after platinum-based chemotherapy.
Category: Cancer
Trade Name (Agent): CLN-081
Sponsor: Cullinan Oncology, Inc.
Date of BT Designation Disclosure: 1/04/2022
Indication: Treatment of patients with locally advanced or metastatic non-small cell lung cancer (“NSCLC”) harboring epidermal growth factor (“EGFR”) exon 20 insertion mutations who have previously received platinum-based systemic chemotherapy.
Category: Cancer
Trade Name (Agent): Vedotin (Teliso-V)
Sponsor: AbbVie Inc.
Date of BT Designation Disclosure: 1/04/2022
Indication: Treatment of patients with advanced/metastatic epidermal growth factor receptor (EGFR) wild type, nonsquamous non-small cell lung cancer (NSCLC) with high levels of c-Met overexpression whose disease has progressed on or after platinum-based therapy.
Category: Cancer
Trade Name (Agent): Patritumab Deruxtecan (HER3-DXd)
Sponsor: Daiichi Sankyo Company, Ltd.
Date of BT Designation Disclosure: 12/23/2021
Indication: Treatment of patients with metastatic or locally advanced EGFR-mutated non-small cell lung cancer (NSCLC) with disease progression on or after treatment with a third-generation tyrosine kinase inhibitor (TKI) and platinum-based therapies.
Category: Cancer
Trade Name (Agent): Aficamten
Sponsor: Cytokinetics, Inc.
Date of BT Designation Disclosure: 12/09/2021
Indication: Treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM).
Category: Cardiovascular
Trade Name (Agent): Gantenerumab
Sponsor: GENENTECH INC
Date of BT Designation Disclosure: 10/08/2021
Indication: Treatment of people living with Alzheimer’s disease (AD).
Category: Other
Trade Name (Agent): Tecartus (brexucabtagene autoleucel)
Sponsor: Kite Pharma Inc.
Date of BT Designation Disclosure: 2017
Approval Date: 10/01/2021
Indication: Treatment of adult patients (18 years and older) with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL).
Category: Cancer
Trade Name (Agent): Repotrectinib
Sponsor: Zai Lab Limited and Turning Point Therapeutics, Inc.
Date of BT Designation Disclosure: 10/04/2021
Indication: Treatment of patients with advanced solid tumors that have an NTRK gene fusion who have progressed following treatment with one or two prior TRK tyrosine kinase inhibitors, with or without prior chemotherapy, and have no satisfactory alternative treatment
Category: Cancer
Trade Name (Agent): ENHERTU (fam-trastuzumab deruxtecan-nxki)
Sponsor: AstraZeneca, Daiichi Sankyo
Date of BT Designation Disclosure: 10/04/2021
Indication: Treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received one or more prior anti-HER2-based regimens.
Category: Cancer
Trade Name (Agent): EXKIVITY (mobocertinib)
Sponsor: Takeda Pharmaceutical Company Ltd.
Date of BT Designation Disclosure: 4/27/2020
Approval Date: 9/15/2021
Indication: Treatment of NSCLC Patients with EGFR Exon 20 Insertion Mutations
Category: Cancer
Trade Name (Agent): JARDIANCE (empagliflozin)
Sponsor: Eli Lilly and Co.
Date of BT Designation Disclosure: 9/09/2021
Indication: Investigational treatment of heart failure with preserved ejection fraction
Category: Cardiovascular
Trade Name (Agent): Toripalimab in combination with Gemcitabine and Cisplatin
Sponsor: Shanghai Junshi Biosciences Co., Ltd. and Coherus BioSciences, Inc.
Date of BT Designation Disclosure: 8/12/2021
Indication: First-line treatment of recurrent or metastatic nasopharyngeal carcinoma
Category: Cancer
Trade Name (Agent): Nexviazyme (avalglucosidase alfa-ngpt)
Sponsor: Sanofi/Genzyme
Date of BT Designation Disclosure: 11/18/2020
Approval Date: 8/06/2021
Indication: Treatment of patients one year of age and older with late-onset Pompe disease.
Category: Other
Trade Name (Agent): Avexitide
Sponsor: Eiger BioPharmaceuticals Inc.
Date of BT Designation Disclosure: 8/05/2021
Indication: Treatment of congenital hyperinsulism (HI).
Category: Other
Trade Name (Agent): ARO-AAT
Sponsor: Arrowhead Pharmaceuticals Inc.
Date of BT Designation Disclosure: 7/29/2021
Indication: Treatment for the rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD).
Category: Rare Inherited Disorders
Trade Name (Agent): TAK-994
Sponsor: Takeda Pharmaceutical Company Ltd.
Date of BT Designation Disclosure: 7/28/2021
Indication: Treatment of excessive daytime sleepiness (EDS) in patients with narcolepsy type 1 (NT1).
Category: Other
Trade Name (Agent): Venclexta (venetoclax)
Sponsor: GENENTECH INC
Date of BT Designation Disclosure: 7/21/2021
Indication: Treatment of adult patients with previously untreated intermediate, high- and very high-risk myelodysplastic syndromes (MDS) based on the revised International Prognostic Scoring System (IPSS-R).
Category: Other
Trade Name (Agent): ALK-001 (C20-D3-vitamin A)
Sponsor: Alkeus Pharmaceuticals Inc.
Date of BT Designation Disclosure: 7/14/2021
Indication: Treatment of Stargardt Disease.
Category: Other
Trade Name (Agent): ASP3772
Sponsor: Affinivax Inc., Astellas Pharma US, Inc.
Date of BT Designation Disclosure: 7/13/2021
Indication: Vaccine candidate targeting Streptococcus pneumoniae designed to offer both B-cell (antibody) and T-cell immune protection.
Category: Infectious Disease
Trade Name (Agent): VLA1553
Sponsor: Valneva SE
Date of BT Designation Disclosure: 7/07/2021
Indication: Chikungunya Vaccine
Category: Infectious Disease
Trade Name (Agent): Orelabrutinib
Sponsor: InnoCare Pharma Ltd.
Date of BT Designation Disclosure: 6/28/2021
Indication: Treatment of relapsed or refractory mantle cell lymphoma (R/R MCL).
Category: Cancer
Trade Name (Agent): Adagrasib
Sponsor: Mirata Therapeutics Inc.
Date of BT Designation Disclosure: 6/25/2021
Indication: Treatment of non-small cell lung cancer (NSCLC) in patients harboring the KRASG12C mutation after systemic therapy.
Category: Cancer
Trade Name (Agent): Donanemab
Sponsor: Eli Lilly and Co.
Date of BT Designation Disclosure: 6/25/2021
Indication: Investigational antibody therapy for Alzheimer's disease (AD).
Category: Other
Trade Name (Agent): Lecanemab (BAN2401)
Sponsor: Eisai Co., Eisai Co. and Biogen Inc.
Date of BT Designation Disclosure: 6/23/2021
Indication: Investigational anti-amyloid beta (Aβ) protofibril antibody for the treatment of Alzheimer's disease.
Category: Other
Trade Name (Agent): Pluvicto (Lu-PSMA-617)
Sponsor: Novartis Pharmaceuticals Corporation
Date of BT Designation Disclosure: 6/16/2021
Approval Date: 4/23/2022
Indication: Treatment for advanced castration-resistant prostate cancer.
Category: Cancer
Trade Name (Agent): Ayvakit (Avapritinib)
Sponsor: Blueprint Medicines Corporation
Date of BT Designation Disclosure: 1/12/2021
Approval Date: 6/16/2021
Indication: Treatment for patients with advanced systemic mastocytosis (AdvSM), including patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL).
Category: Other
Trade Name (Agent): VS-6766 in combination with Defactinib
Sponsor: Verastem, Inc
Date of BT Designation Disclosure: 5/24/2021
Indication: Treatment of all patients with recurrent low-grade serous ovarian cancer (LGSOC) regardless of KRAS status after one or more prior lines of therapy, including platinum-based chemotherapy.
Category: Cancer
Trade Name (Agent): Rusfertide
Sponsor: Protagonist Therapeutics, Inc.
Date of BT Designation Disclosure: 6/03/2021
Indication: Treatment of patients with polycythemia vera (PV) for the reduction of erythrocytosis in those patients who do not require further treatment for thrombocytosis and/or leukocytosis.
Category: Other
Trade Name (Agent): Teclistamab
Sponsor: Janssen Pharmaceuticals Inc.
Date of BT Designation Disclosure: 6/01/2021
Indication: Treatment for patients with relapsed/refractory multiple myeloma.
Category: Cancer
Trade Name (Agent): Bemarituzumab
Sponsor: Amgen Inc.
Date of BT Designation Disclosure: 4/19/2021
Indication: Treatment for patients with fibroblast growth factor receptor 2b (FGFR2b) overexpressing and human epidermal growth factor receptor 2 (HER2)-negative metastatic and locally advanced gastric and gastroesophageal (GEJ) adenocarcinoma in combination with mod
Category: Cancer
Trade Name (Agent): TTP399
Sponsor: vTv Therapeutics Inc.
Date of BT Designation Disclosure: 4/13/2021
Indication: Adjunctive therapy to insulin for the treatment of type 1 diabetes
Category: Other
Trade Name (Agent): Futibatinib (TAS-120)
Sponsor: Taiho Oncology, Inc. and Taiho Pharmaceutical Co., Ltd
Date of BT Designation Disclosure: 4/01/2021
Indication: Treatment of patients with previously treated locally advanced or metastatic cholangiocarcinoma harboring FGFR2 gene rearrangements, including gene fusions
Category: Cancer
Trade Name (Agent): Parsaclisib (IBI376)
Sponsor: Innovent Biologics, Inc.
Date of BT Designation Disclosure: 3/21/2021
Indication: Treatment of patients with relapsed/refractory follicular lymphoma (FL). IBI376 is a phosphatidylinositol 3-kinase delta (PI3Kδ) inhibitor originally discovered by Incyte. Innovent owns the rights to develop and commercialize Parsaclisib in greater China.
Category: Cancer
Trade Name (Agent): BXCL501
Sponsor: BioXcel Therapeutics, Inc.
Date of BT Designation Disclosure: 3/15/2021
Indication: Acute treatment of agitation associated with dementia
Category: Other
Trade Name (Agent): Nulibry (fosdenopterin)
Sponsor: BridgeBio Pharma, Inc.
Date of BT Designation Disclosure: 2/26/2021
Approval Date: 2/26/2021
Indication: Treatment for molybdenum Cofactor Deficiency (MoCD) Type A
Category: Rare Inherited Disorders
Trade Name (Agent): Cabozantinib (CABOMETYX®)
Sponsor: Exelixis Inc.
Date of BT Designation Disclosure: 2/25/2021
Approval Date: 9/17/2021
Indication: Treatment for patients with differentiated thyroid cancer (DTC) that has progressed following prior therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate).
Category: Cancer
Trade Name (Agent): Tipifarnib
Sponsor: Kura Oncology, Inc.
Date of BT Designation Disclosure: 2/24/2021
Indication: Treatment of patients with recurrent or metastatic HRAS mutant head and neck squamous cell carcinoma (HNSCC) with variant allele frequency ≥ 20% after disease progression on platinum-based chemotherapy.
Category: Cancer
Trade Name (Agent): Tebentafusp (IMCgp100)
Sponsor: Immunocore
Date of BT Designation Disclosure: 2/19/2021
Approval Date: 1/25/2022
Indication: Treatment of HLA-A*02:01-positive adult patients with unresectable or metastatic uveal melanoma (mUM).
Category: Cancer
Trade Name (Agent): Scemblix (Asciminib)
Sponsor: Novartis, Novartis International AG
Date of BT Designation Disclosure: 2/08/2021
Approval Date: 10/29/2021
Indication: Treatment of adult patients with Ph+ CML in CP harboring the T315I mutation.
Category: Cancer
Trade Name (Agent): Scemblix (Asciminib)
Sponsor: Novartis, Novartis International AG
Date of BT Designation Disclosure: 2/08/2021
Approval Date: 10/29/2021
Indication: Treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with two or more tyrosine kinase inhibitors (TKIs).
Category: Cancer
Trade Name (Agent): Enhertu (trastuzumab deruxtecan)
Sponsor: AstraZeneca, AstraZeneca plc and Daiichi Sankyo Co.
Date of BT Designation Disclosure: 5/11/2020
Approval Date: 1/15/2021
Indication: Treatment of adult patients with previously treated HER2 positive advanced gastric cancer
Category: Cancer
Trade Name (Agent): DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj)
Sponsor: Janssen Biotech Inc.
Date of BT Designation Disclosure: 2013
Approval Date: 1/15/2021
Indication: Treatment of adult patients with newly diagnosed light-chain (AL) amyloidosis.
Category: Other
Trade Name (Agent): Ligelizumab (QGE031)
Sponsor: Novartis, Novartis International AG
Date of BT Designation Disclosure: 1/14/2021
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
Trade Name (Agent): APL-106 (uproleselan)
Sponsor: Apollomics, Inc
Date of BT Designation Disclosure: 1/07/2021
Indication: Treatment of relapsed/refractory acute myeloid leukemia (AML).
Category: Cancer
Trade Name (Agent): Chiauranib
Sponsor: Shenzhen Chipscreen Biosciences Co., Ltd
Date of BT Designation Disclosure: 1/05/2021
Indication: "Treatment of patients with small cell lung cancer (SCLC) with disease progression or recurrence after two lines of prior systematic chemotherapy. "
Category: Cancer
Trade Name (Agent): Tiragolumab in combination with Tecentriq
Sponsor: The Roche Group
Date of BT Designation Disclosure: 1/05/2021
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
Trade Name (Agent): CNCT19 (CD19 CAR-T)
Sponsor: CASI Pharmaceuticals, Inc.
Date of BT Designation Disclosure: 12/23/2020
Indication: A potential CD19 CAR-T therapy for the treatment of adults with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL)
Category: Cancer
Trade Name (Agent): IO102 and IO103 with anti-PD-1 mAb
Sponsor: IO Biotech
Date of BT Designation Disclosure: 12/15/2020
Indication: Patients with unresectable/ metastatic melanoma
Category: Cancer
Trade Name (Agent): IO102 and IO103 with anti-PD-1 mAb
Sponsor: IO Biotech
Date of BT Designation Disclosure: 12/15/2020
Indication: Patients with unresectable/ metastatic melanoma
Category: Cancer
Trade Name (Agent): CUTX-101 (Copper Histidinate)
Sponsor: Cyprium Therapeutics, Inc.
Date of BT Designation Disclosure: 12/15/2020
Indication: Treatment of Menkes Disease
Category: Rare Inherited Disorders
Trade Name (Agent): Blenrep (BELANTAMAB MAFODOTIN-BLMF)
Sponsor: GlaxoSmithKline Intellectual Property Development LTD England
Date of BT Designation Disclosure: 11/02/2017
Approval Date: 8/05/2020
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
Trade Name (Agent): Avastin (Bevacizumab)
Sponsor: GENENTECH INC
Date of BT Designation Disclosure: 7/18/2018
Approval Date: 5/29/2020
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
