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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 August 31, 2021, the FDA has approved 218* breakthrough therapy designated products and lists that there have been 1182 total requests for the designation with 458** designations granted.

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Trade Name (Agent)

Sponsor

Date of BT Designation Disclosure

Approval Date

Indication

Category

Trade Name (Agent): Talquetamab

Sponsor: Janssen Pharmaceuticals Inc.

Date of BT Designation Disclosure: 6/29/2022

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 and 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 plc and Daiichi Sankyo Co.

Date of BT Designation Disclosure: 4/26/2022

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): Dapagliflozin (Farxiga®)

Sponsor: AstraZeneca plc

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

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 plc and Daiichi Sankyo Co.

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. and Astellas Pharma 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. 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): Lu-PSMA-617

Sponsor: Novartis Pharmaceuticals Corporation

Date of BT Designation Disclosure: 6/16/2021

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

Indication: Treatment of HLA-A*02:01-positive adult patients with unresectable or metastatic uveal melanoma (mUM).

Category: Cancer

Trade Name (Agent): Asciminib (ABL001)

Sponsor: Novartis International AG

Date of BT Designation Disclosure: 2/08/2021

Indication: Treatment of adult patients with Ph+ CML in CP harboring the T315I mutation.

Category: Cancer

Trade Name (Agent): Asciminib (ABL001)

Sponsor: Novartis International AG

Date of BT Designation Disclosure: 2/08/2021

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 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 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

Trade Name (Agent): Pegloprastide (AVB-620)

Sponsor: Avelas Biosciences

Date of BT Designation Disclosure: 12/14/2020

Indication: Intraoperative detection and visualization of positive margins during breast cancer surgery.

Category: Cancer

Trade Name (Agent): Zanidatamab

Sponsor: Zymeworks Inc.

Date of BT Designation Disclosure: 11/30/2020

Indication: Treatment for patients with previously-treated HER2 gene-amplified biliary tract cancer (BTC).

Category: Cancer

Trade Name (Agent): Zanidatamab

Sponsor: Zymeworks Inc.

Date of BT Designation Disclosure: 11/30/2020

Indication: Treatment for patients with previously-treated HER2 gene-amplified biliary tract cancer (BTC).

Category: Cancer

Trade Name (Agent): Sotorasib

Sponsor: Amgen

Date of BT Designation Disclosure: 12/08/2020

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

Trade Name (Agent): Repotrectinib

Sponsor: Turning Point Therapeutics Inc.

Date of BT Designation Disclosure: 12/08/2020

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

Trade Name (Agent): Olorofim

Sponsor:  F2G Ltd

Date of BT Designation Disclosure: 10/22/2020

Indication: Treatment of Central Nervous System (CNS) coccidioidomycosis refractory or otherwise unable to be treated with standard of care therapy

Category: Infectious Disease

Trade Name (Agent): Vixarelimab

Sponsor: Kiniksa Pharmaceuticals Ltd

Date of BT Designation Disclosure: 11/16/2020

Indication: Treatment of pruritus associated with prurigo nodularis, a chronic inflammatory skin condition characterized by severely pruritic skin nodules

Category: Other

Trade Name (Agent): Inmazeb (atoltivimab, maftivimab, and odesivimab-ebgn)

Sponsor: Regeneron Pharmaceuticals

Date of BT Designation Disclosure: 11/19/2019

Approval Date: 10/14/2020

Indication: Treatment for Zaire ebolavirus (Ebola virus) infection in adult and pediatric patients

Category: Infectious Disease

Trade Name (Agent): EBANGA ANSUVIMAB-ZYKL

Sponsor: Ridgeback Biotherapeutics LP

Date of BT Designation Disclosure: 9/06/2019

Approval Date: 12/21/2020

Indication: Treatment for Ebola

Category: Infectious Disease

Trade Name (Agent): Lanifibranor

Sponsor: Inventiva

Date of BT Designation Disclosure: 10/12/2020

Indication: Treatment of non-alcoholic steatohepatitis (NASH), mucopolysaccharidoses (MPS)

Category: Other

Trade Name (Agent): IMGN632

Sponsor: ImmunoGen, Inc

Date of BT Designation Disclosure: 10/05/2020

Indication: Treatment of relapsed or refractory blastic plasmacytoid dendritic cell neoplasm

Category: Cancer

Trade Name (Agent): Farxiga® (dapagliflozin)

Sponsor: AstraZeneca 

Date of BT Designation Disclosure: 10/02/2020

Approval Date: 4/30/2021

Indication: Patients with chronic kidney disease (CKD), with and without type 2 diabetes (T2D)

Category: Other

Trade Name (Agent): BI 1482694

Sponsor: Boehringer Ingelheim

Date of BT Designation Disclosure: 12/18/2015

Indication: Advanced/pre-treated EGFR mutation-positive NSCLC

Category: Cancer

Trade Name (Agent): Pembrolizumab (Keytruda)

Sponsor: Merck

Date of BT Designation Disclosure: 11/02/2015

Indication: Microsatellite Instability-High (MSI-H) metastatic colorectal cancer (mCRC)

Category: Cancer

Trade Name (Agent): Inotuzumab Ozogamicin

Sponsor: Pfizer

Date of BT Designation Disclosure: 10/19/2015

Indication: ALL

Category: Cancer

Trade Name (Agent): RBX2660

Sponsor: Rebiotix, Inc.

Date of BT Designation Disclosure: 10/12/2015

Indication: Recurrent Clostridium difficile (C diff) infection

Category: Infectious Disease

Trade Name (Agent): BMS-663068

Sponsor: BMS

Date of BT Designation Disclosure: 7/21/2015

Indication: HIV-1 in heavily treatment-experienced adult patients

Category: Infectious Disease

Trade Name (Agent): DX-2930 

Sponsor: Dyax

Date of BT Designation Disclosure: 7/07/2015

Indication: Hereditary angioedema (HAE)

Category: Rare Inherited Disorders

Trade Name (Agent): VTS 270

Sponsor: Vtesse

Date of BT Designation Disclosure: 1/06/2016

Indication: Niemann-Pick Type C1 Disease (NPC)

Category: Rare Inherited Disorders

Trade Name (Agent): Lynparza (olaparib)

Sponsor: AstraZeneca

Date of BT Designation Disclosure: 1/27/2016

Indication: Metastatic Castration Resistant Prostate Cancer

Category: Cancer

Trade Name (Agent): Venetoclax

Sponsor: AbbVie/Roche

Date of BT Designation Disclosure: 1/28/2016

Indication: AML combination therapy

Category: Cancer

Trade Name (Agent): Rapastinel (GLYX-13)

Sponsor: Allergan

Date of BT Designation Disclosure: 1/31/2016

Indication: Major Depressive Disorder (MDD)

Category: Other

Trade Name (Agent): Affinity enhanced T-cell therapy

Sponsor: Adaptimmune

Date of BT Designation Disclosure: 2/09/2016

Indication: NY-ESO in synovial sarcoma

Category: Cancer

Trade Name (Agent): Durvalumab (MEDI4736)

Sponsor: AstraZeneca

Date of BT Designation Disclosure: 2/17/2016

Indication: PD-L1+ inoperable/metastatic urothelial bladder cancer

Category: Cancer

Trade Name (Agent): PKC412 (midostaurin)

Sponsor: Novartis

Date of BT Designation Disclosure: 2/19/2016

Indication: Newly-diagnosed FLT3-mutated AML

Category: Cancer

Trade Name (Agent): NI-0501

Sponsor: Novimmune

Date of BT Designation Disclosure: 3/16/2016

Indication: Primary Hemophagocytic Lymphohistiocytosis (HLH)

Category: Rare Inherited Disorders

Trade Name (Agent): SER-109

Sponsor: Seres

Date of BT Designation Disclosure: 6/12/2015

Indication: Clostridium difficile infection (CDI)

Category: Infectious Disease

Trade Name (Agent): ACTEMRA (tocilizumab)

Sponsor: Genentech/Roche

Date of BT Designation Disclosure: 6/10/2015

Approval Date: 3/04/2021

Indication: Systemic scerlosis

Category: Other

Trade Name (Agent): MYDICAR

Sponsor: Celladon 

Date of BT Designation Disclosure: 4/10/2014

Indication: Heart failure

Category: Cardiovascular

Trade Name (Agent): Rociletinib (CO-1686)

Sponsor: Clovis Oncology

Date of BT Designation Disclosure: 5/19/2014

Indication: NSCLC

Category: Cancer

Trade Name (Agent): CRS-207 and GVAX

Sponsor: Aduro

Date of BT Designation Disclosure: 7/21/2014

Indication: Pancreatic cancer

Category: Cancer

Trade Name (Agent): AP26113

Sponsor: Ariad

Date of BT Designation Disclosure: 10/02/2014

Indication: ALK-positive NSCLC

Category: Cancer

Trade Name (Agent): SPK-RPE65

Sponsor: Spark Therapeutics

Date of BT Designation Disclosure: 11/06/2014

Indication: Inherited retinal dystrophy (IRD)

Category: Rare Inherited Disorders

Trade Name (Agent): JCAR015

Sponsor: Juno Therapeutics

Date of BT Designation Disclosure: 11/24/2014

Indication: ALL

Category: Cancer