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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 251* 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): Zydelig (idelalisib)

Sponsor: Gilead Sciences Inc.

Date of BT Designation Disclosure: 11/18/2013

Approval Date: 7/23/2014

Indication: Patients with relapsed chronic lymphocytic leukemia, in combination with rituximab, in patients for whom rituximab alone would be considered appropriate therapy due to other co-morbidities

Category: Cancer

Trade Name (Agent): Arzerra (ofatumumab)

Sponsor: Novartis Pharmaceuticals Corp.

Date of BT Designation Disclosure: 9/13/2013

Approval Date: 4/17/2014

Indication: Previously untreated patients with chronic lymphocytic leukemia (CLL) for whom fludarabine-based therapy is considered inappropriate

Category: Cancer

Trade Name (Agent): Gazyva (obinutuzumab)

Sponsor: GENENTECH INC

Date of BT Designation Disclosure: 5/15/2013

Approval Date: 11/01/2013

Indication: Patients with previously untreated chronic lymphocytic leukemia in combination with chlorambucil

Category: Cancer

Trade Name (Agent): Braftovi (encorafenib)

Sponsor: Array BioPharma Inc.

Date of BT Designation Disclosure: 2019

Approval Date: 4/08/2020

Indication: In combination with cetuximab for the treatment of adult patients with metastatic colorectal cancer (CRC) with a BRAF V600E mutation, detected by an FDA-approved test, after prior therapy

Category: Cancer

Trade Name (Agent): Exebacase

Sponsor: ContraFect Corporation

Date of BT Designation Disclosure: 2/24/2020

Indication: Patients with MRSA bacteremia, including right-sided endocarditis, when used in conjunction with standard of care antibiotics

Category: Infectious Disease

Trade Name (Agent): Cibinqo (Abrocitinib)

Sponsor: Pfizer Inc.

Date of BT Designation Disclosure: 2/14/2018

Approval Date: 1/14/2022

Indication: Treatment of adults with refractory, moderate-to-severe atopic dermatitis whose disease is not adequately controlled with other systemic drug products, including biologics, or when use of those therapies is inadvisable

Category: Other

Trade Name (Agent): Keytruda (pembrolizumab)

Sponsor: Merck Sharpe & Dohme Corp.

Date of BT Designation Disclosure: 7/15/2019

Indication: Patients with hepatocellular carcinoma

Category: Cancer

Trade Name (Agent): Harvoni (sofosbuvir/ledipasvir)

Sponsor: Gilead Sciences Inc.

Date of BT Designation Disclosure: 7/25/2013

Approval Date: 10/10/2014

Indication: Patients with chronic hepatitis C, genotype 1 infection

Category: Infectious Disease

Trade Name (Agent): Ofev (nintedanib)

Sponsor: Boehringer Ingelheim, Boehringer Ingelheim Pharmaceuticals Inc.

Date of BT Designation Disclosure: 7/16/2014

Approval Date: 10/15/2014

Indication: Patients with idiopathic pulmonary fibrosis (IPF)

Category: Other

Trade Name (Agent): Esbriet (pirfenidone)

Sponsor: GENENTECH INC

Date of BT Designation Disclosure: 7/17/2014

Approval Date: 10/15/2014

Indication: Patients with idiopathic pulmonary fibrosis (IPF)

Category: Other

Trade Name (Agent): Technivie (ombitasvir, paritaprevir, ritonavir)

Sponsor: AbbVie Inc.

Date of BT Designation Disclosure: 6/30/2014

Approval Date: 7/24/2015

Indication: Patients with genotype 4 chronic hepatitis C viruse (HCV) infection without cirrhosis

Category: Infectious Disease

Trade Name (Agent): Orkambi (lumacaftor and ivacaftor)

Sponsor: Vertex Pharmaceuticals Inc.

Date of BT Designation Disclosure: 1/29/2014

Approval Date: 7/02/2015

Indication: Patients 12 years and older with cystic fibrosis who are homozygous for the F508del mutation in the CFTR gene

Category: Rare Inherited Disorders

Trade Name (Agent): Eylea (aflibercept)

Sponsor: Regeneron Pharmaceuticals Inc.

Date of BT Designation Disclosure: 9/16/2014

Approval Date: 3/25/2015

Indication: Treatment of diabetic retinopathy (DR) in patients with diabetic macular edema (DME)

Category: Other

Trade Name (Agent): Kalydeco (ivacaftor)

Sponsor: Vertex Pharmaceuticals Inc.

Date of BT Designation Disclosure: 12/15/2012

Approval Date: 3/17/2015

Indication: Patients 2 years and older with cystic fibrosis and have that one of the following CFTR gene mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, R117H

Category: Rare Inherited Disorders

Trade Name (Agent): Lucentis (ranibizumab)

Sponsor: GENENTECH INC

Date of BT Designation Disclosure: 12/15/2014

Approval Date: 2/06/2015

Indication: Treatment of Diabetic Retinopathy (DR) in patients with Diabetic Macula Edema (DME)

Category: Other

Trade Name (Agent): Xuriden (uridine triacetate)

Sponsor: Wellstat Therapeutics Corp.

Date of BT Designation Disclosure: 4/30/2014

Approval Date: 9/04/2015

Indication: Patients with hereditary orotic aciduria

Category: Rare Inherited Disorders

Trade Name (Agent): Blincyto (blinatumomab)

Sponsor: Amgen Inc.

Date of BT Designation Disclosure: 7/01/2014

Approval Date: 12/03/2014

Indication: Patients with Philadelphia chromosome-negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL)

Category: Cancer

Trade Name (Agent): Viekira Pak (ombitasvir, paritaprevir, ritonavir)

Sponsor: AbbVie Inc.

Date of BT Designation Disclosure: 5/06/2013

Approval Date: 12/19/2014

Indication: Patients with genotype 1 chronic hepatitis C virus (HCV) infection including those with compensated cirrhosis

Category: Infectious Disease

Trade Name (Agent): Opdivo (nivolumab)

Sponsor: Bristol-Myers Squibb Company

Date of BT Designation Disclosure: 2/15/2013

Approval Date: 12/22/2014

Indication: Patients with unresectable or metastatic melanoma and disease progression following ipilimumab and, if BRAF V600 mutation positive, a BRAF inhibitor

Category: Cancer

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 US, Inc.

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): Olumiant (Baricitinib)

Sponsor: Eli Lilly and Company + Incyte Corporation

Date of BT Designation Disclosure: 3/16/2020

Approval Date: 6/13/2022

Indication: Treatment of adult patients with severe 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, 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: 11/11/2019

Approval Date: 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, 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

Approval Date: 4/28/2022

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