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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 December 31, 2023, the FDA has approved 295* breakthrough therapy designated products and lists that there have been 1438 total requests for the designation with 544** designations granted.

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Trade Name (Agent)

Sponsor

Date of BT Designation Disclosure

Approval Date

Indication

Category

Trade Name (Agent): Tibsovo (ivosidenib)

Sponsor: Servier Pharmaceuticals

Date of BT Designation Disclosure: 3/26/2019

Approval Date: 10/24/2023

Indication: Treatment for adult patients with relapsed or refractory (R/R) myelodysplastic syndromes (MDS) with an isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test.

Category:

Trade Name (Agent): Nipocalimab

Sponsor: J&J

Date of BT Designation Disclosure: 2/9/2024

Indication: Treatment for individuals at high risk for severe hemolytic disease of the fetus and newborn (HDFN)

Category: Rare Inherited Disorders

Trade Name (Agent): Latozinemab

Sponsor: Alector Inc.

Date of BT Designation Disclosure: 2/7/2024

Indication: An investigational human monoclonal antibody designed to block sortilin to elevate progranulin (PGRN) levels for the potential treatment of frontotemporal dementia with a progranulin gene mutation (FTD-GRN)

Category: Other

Trade Name (Agent): PF614-MPAR

Sponsor: Ensysce Biosciences

Date of BT Designation Disclosure: 1/29/2024

Indication: To provide optimal pain relief at prescribed doses yet limit accidental or intentional overdose by ‘shutting down' opioid release if too much active drug is consumed.

Category:

Trade Name (Agent): Balversa (erdafitinib)

Sponsor: J&J

Date of BT Designation Disclosure: 2018

Approval Date: 1/19/2024

Indication: Treatment for adult patients with locally advanced or metastatic urothelial carcinoma (mUC) with susceptible FGFR3 genetic alterations, as determined by an FDA-approved companion diagnostic test, whose disease has progressed on or after at least one line of prior systemic therapy.

Category: Cancer

Trade Name (Agent): Cretostimogene Grenadenorepvec

Sponsor: CG Oncology

Date of BT Designation Disclosure: 1/3/2024

Indication: Potential therapeutic option in patients with high-risk BCG-unresponsive non–muscle invasive bladder cancer with carcinoma in situ with or without Ta or T1 tumors.

Category: Cancer

Trade Name (Agent): Xolair (omalizumab)

Sponsor: Roche

Date of BT Designation Disclosure: 8/12/2018

Indication: Prevention of severe allergic reactions following accidental exposure to one or more foods in people with allergies.

Category: Other

Trade Name (Agent): mRNA-1345

Sponsor: Moderna

Date of BT Designation Disclosure: 1/3/2023

Indication: Prevention of RSV-associated lower respiratory tract disease (RSV-LRTD) in adults aged 60 years or older.

Category: Infectious Disease

Trade Name (Agent): Enhertu (fam-trastuzumab-deruxtecan-nxki)

Sponsor: AstraZeneca and Daiichi Sankyo Co.

Date of BT Designation Disclosure: 8/21/2023

Indication: Treatment for patients with HER2-positive metastatic colorectal cancer (mCRC) who have received at least 2 prior lines of therapy.

Category: Cancer

Trade Name (Agent): Enhertu (fam-trastuzumab-deruxtecan-nxki)

Sponsor: AstraZeneca and Daiichi Sankyo Co.

Date of BT Designation Disclosure: 8/21/2023

Indication: Treatment of patients with unresectable or metastatic HER2-positive solid tumors that have progressed after prior treatment and who have no satisfactory alternative treatment options

Category: Cancer

Trade Name (Agent): Epkinly™ (epcoritamab-bysp)

Sponsor: AbbVie Inc.

Date of BT Designation Disclosure: 11/27/2023

Indication: Treatment of Relapsed/Refractory Follicular Lymphoma

Category: Cancer

Trade Name (Agent): BMS-986278

Sponsor: Bristol-Myers Squibb Co.

Date of BT Designation Disclosure: 10/24/2023

Indication: A potential first-in-class, oral, lysophosphatidic acid receptor 1 (LPA1) antagonist, for the treatment of progressive pulmonary fibrosis (PPF)

Category: Other

Trade Name (Agent): AST2818 (furmonertinib)

Sponsor: ArriVent Biopharma

Date of BT Designation Disclosure: 10/30/2023

Indication: For use as a potential therapeutic option in patients with previously untreated, locally advanced or metastatic nonsquamous non–small cell lung cancer harboring EGFR exon 20 insertion mutations.

Category: Cancer

Trade Name (Agent): Eflornithine (DFMO)

Sponsor: US WorldMeds LLC

Date of BT Designation Disclosure: 4/3/2020

Approval Date: 12/13/2023

Indication: To reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (HRNB) who have demonstrated at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy.

Category: Cancer

Trade Name (Agent): Keytruda (pembrolizumab) + Padcev (enfortumab vedotin-ejfv)

Sponsor: Astellas Pharma US, Inc., Merck & Co., Inc.

Date of BT Designation Disclosure: 4/3/2023

Approval Date: 12/15/2023

Indication: Treatment for patients with locally advanced or metastatic urothelial cancer (la/mUC).

Category: Cancer

Trade Name (Agent): WELIREG™ (belzutifan)

Sponsor: Merck & Co., Inc.

Date of BT Designation Disclosure: 12/14/2023

Indication: Treatment for patients with renal cell carcinoma who have received prior treatment.

Category: Cancer

Trade Name (Agent): VT-X7

Sponsor: Osteal Therapeutics

Date of BT Designation Disclosure: 12/5/2023

Indication: A drug-device combination for periprosthetic joint infection in hip and knee replacement patients

Category: Other

Trade Name (Agent): Crinecerfont

Sponsor: Neurocrine Biosciences Inc.

Date of BT Designation Disclosure: 12/6/2023

Indication: Treatment of congenital adrenal hyperplasia

Category: Other

Trade Name (Agent): Cretostimogene Grenadenorepvec

Sponsor: CG Oncology

Date of BT Designation Disclosure: 12/5/2023

Indication: Immunotherapy for high-risk Bacillus Calmette-Guérin (BCG)-unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC) with carcinoma in situ with or without Ta or T1 (papillary) tumors.

Category: Cancer

Trade Name (Agent): Pegozafermin

Sponsor: 89bio

Date of BT Designation Disclosure: 12/4/2023

Indication: Treatment of nonalcoholic steatohepatitis (NASH) with fibrosis

Category: Other

Trade Name (Agent): TAR-200

Sponsor: J&J

Date of BT Designation Disclosure: 12/4/2023

Indication: Potential future treatment of patients with Bacillus Calmette-Guérin (BCG)-unresponsive high-risk non-muscle-invasive bladder cancer (HR-NMIBC), who are ineligible for or elected not to undergo radical cystectomy (surgical removal of the bladder)

Category: Cancer

Trade Name (Agent): Opfolda™ (miglustat)

Sponsor: Amicus Therapeutics

Date of BT Designation Disclosure: 2019

Approval Date: 9/28/2023

Indication: In combination with Opfolda, for the treatment of adult patients with late onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT)

Category: Rare Inherited Disorders

Trade Name (Agent): Pombiliti™ (cipaglucosidase alfa-atga)

Sponsor: Amicus Therapeutics

Date of BT Designation Disclosure: 2019

Approval Date: 9/28/2023

Indication: In combination with Opfolda, for the treatment of adult patients with late onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT)

Category: Rare Inherited Disorders

Trade Name (Agent): Pegozafermin

Sponsor: 89bio

Date of BT Designation Disclosure: 9/21/2023

Indication: Nonalcoholic Steatohepatitis (NASH)

Category: Other

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

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