Trade Name (Agent): Atacicept
Sponsor: Vera Therapeutics
Date of BT Designation Disclosure: 5/28/2024
Indication: Treatment of IgA nephropathy (IgAN).
Category: Rare Inherited Disorders
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 June 30, 2024, the FDA has approved 317* breakthrough therapy designated products and lists that there have been 1516 total requests for the designation with 587 designations granted.
Trade Name (Agent)
Sponsor
Date of BT Designation Disclosure
Approval Date
Indication
Category
Trade Name (Agent): inavolisib (GDC-0077) + palbociclib (Ibrance) and fulvestrant
Sponsor: Roche
Date of BT Designation Disclosure: 5/21/2024
Indication: Treatment of PIK3CA-mutated breast cancer.
Category: Cancer
Trade Name (Agent): Larsucosterol
Sponsor: DURECT Corporation
Date of BT Designation Disclosure: 5/21/2024
Indication: Treatment of individuals with severe alcohol-associated hepatitis (AH), an acute form of alcohol-associated liver disease (ALD).
Category: Other
Trade Name (Agent): NVL-655
Sponsor: Nuvalent
Date of BT Designation Disclosure: 5/20/2024
Indication: Treatment of patients with locally advanced or metastatic ALK-positive non-small cell lung cancer (NSCLC) who have been previously treated with two or more ALK tyrosine kinase inhibitors (TKIs).
Category: Cancer
Trade Name (Agent): Delpacibart Etedesiran (AOC 1001)
Sponsor: Avidity Biosciences
Date of BT Designation Disclosure: 5/14/2024
Indication: Investigational treatment for myotonic dystrophy type 1 (DM1)
Category: Rare Inherited Disorders
Trade Name (Agent): Petosemtamab
Sponsor: Merus
Date of BT Designation Disclosure: 5/13/2024
Indication: Treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) who have experienced disease progression following treatment with platinum-based chemotherapy and a PD-L1/PD-1 inhibitor.
Category: Cancer
Trade Name (Agent): Scemblix® (asciminib)
Sponsor: Novartis
Date of BT Designation Disclosure: 5/10/2024
Indication: Treatment of adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP).
Category: Cancer
Trade Name (Agent): Diazoxide Choline
Sponsor: Soleno Therapeutics
Date of BT Designation Disclosure: 4/30/2024
Indication: Treatment of adults and children ages 4 years and older with genetically confirmed Prader-Willi syndrome (PWS) who have hyperphagia.
Category: Rare Inherited Disorders
Trade Name (Agent): Xolremdi (mavorixafor)
Sponsor: X4 Pharmaceuticals
Date of BT Designation Disclosure: 4/29/2024
Indication: Treatment of patients 12 and older with WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis
Category: Rare Inherited Disorders
Trade Name (Agent): BEQVEZ™ (fidanacogene elaparvovec-dzkt)
Sponsor: Pfizer
Date of BT Designation Disclosure: 4/26/2024
Indication: One-Time Gene Therapy for Adults with Hemophilia B
Category: Rare Inherited Disorders
Trade Name (Agent): Anktiva
Sponsor: Immunity Bio
Date of BT Designation Disclosure: 4/22/2024
Indication: Treatment of BCG-Unresponsive Non-Muscle Invasive Bladder Cancer
Category: Cancer
Trade Name (Agent): ENHERTU®
Sponsor: AstraZeneca and Daiichi Sankyo Co.
Date of BT Designation Disclosure: 4/5/2024
Indication: HER2 Directed Therapy for Previously Treated Patients with Metastatic HER2 Positive Solid Tumors
Category: Cancer
Trade Name (Agent): WINREVAIR™ (sotatercept-csrk)
Sponsor: Merck
Date of BT Designation Disclosure: 3/26/2024
Indication: Treatment of Adults with Pulmonary Arterial Hypertension
Category: Other
Trade Name (Agent): Nipocalimab
Sponsor: J&J
Date of BT Designation Disclosure: 3/25/2024
Indication: Treatment of alloimmunized pregnant individuals at high risk of severe hemolytic disease of the fetus and newborn (HDFN)
Category: Other
Trade Name (Agent): CYB003
Sponsor: Cybin
Date of BT Designation Disclosure: 3/14/2024
Indication: Adjunctive treatment for major depressive disorder (MDD)
Category: Other
Trade Name (Agent): Lysergide d-tartrate
Sponsor: Mind Medicine
Date of BT Designation Disclosure: 3/7/2024
Indication: Treatment of generalized anxiety disorder
Category: Other
Trade Name (Agent): Ziftomenib
Sponsor: Kura Oncology, Inc.
Date of BT Designation Disclosure: 4/2/2024
Indication: Treatment of heavily pretreated patients with relapsed/refractory NPM1-mutant acute myeloid leukemia (AML)
Category: Cancer
Trade Name (Agent): Epkinly™ (epcoritamab-bysp)
Sponsor: Genmab U.S.
Date of BT Designation Disclosure: 11/27/2023
Indication: Treatment for adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) who have received 2 or more lines of systemic therapy.
Category: Cancer
Trade Name (Agent): Rivfloza (nedosiran)
Sponsor: Novo Nordisk
Date of BT Designation Disclosure: 7/15/2019
Approval Date: 10/2/2023
Indication: Treatment for primary hyperoxaluria type 1 in patients aged 9 years and older with relatively preserved kidney function
Category: Other
Trade Name (Agent): Dupixent (dupilumab)
Sponsor: Regeneron Pharmaceuticals, Sanofi
Date of BT Designation Disclosure: 8/1/2023
Indication: Respiratory - Add-on maintenance treatment of adults with uncontrolled chronic obstructive pulmonary disease (COPD) associated with a history of exacerbations and an eosinophilic phenotype
Category: Rare Inherited Disorders
Trade Name (Agent): Leqembi (LECANEMAB-IRMB)
Sponsor: Esai
Date of BT Designation Disclosure: 7/2022
Approval Date: 1/6/2023
Indication: Treatment of Alzheimer’s disease
Category: Other
Trade Name (Agent): Tukysa (Tucatinib)
Sponsor: Seagen
Date of BT Designation Disclosure: 7/2022
Approval Date: 1/19/2023
Indication: Treatment for adult patients In combination with trastuzumab with RAS wildtype, HER2-positive, unresectable or metastatic colorectal cancer that has progressed following treatment with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy
Category: Cancer
Trade Name (Agent): Omisirge (omidubicel-onlv)
Sponsor: Gamida Cell
Date of BT Designation Disclosure: 4/17/2023
Indication: Treatment of patients with hematologic malignancies who are in need of a hematopoeitic stem cell transplant.
Category: Rare Inherited Disorders
Trade Name (Agent): VOWST (Fecal Microbiota Spores)
Sponsor: Seres Therapeutics, Inc.
Date of BT Designation Disclosure: 4/26/2023
Indication: To prevent the recurrence of Clostridioides difficile infection (CDI) in individuals 18 years of age and older following antibacterial treatment for recurrent CDI (rCDI).
Category: Infectious Disease
Trade Name (Agent): Roctavian (valoctocogene roxaparvovec-rvox B)
Sponsor: Biomarin Pharmaceutical Inc.
Date of BT Designation Disclosure: 6/29/2023
Indication: For the treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity <1 IU/dL) without pre-existing antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA approved test.
Category: Rare Inherited Disorders
Trade Name (Agent): Abrysvo (Respiratory Syncytial Virus Vaccine)
Sponsor: Pfizer Inc.
Date of BT Designation Disclosure: 3/2/2022
Approval Date: 8/1/2023
Indication: For the prevention of lower respiratory tract disease and severe lower respiratory tract disease caused by respiratory syncytial virus (RSV) in infants from birth through 6 months of age by active immunization of pregnant individuals.
Category: Infectious Disease
Trade Name (Agent): Abrysvo (Respiratory Syncytial Virus Vaccine)
Sponsor: Pfizer Inc.
Date of BT Designation Disclosure: 3/24/2022
Approval Date: 5/31/2023
Indication: For active immunization for the prevention of lower respiratory tract disease (LRTD) caused by respiratory syncytial virus (RSV) in individuals 60 years of age and older
Category: Infectious Disease
Trade Name (Agent): IXCHIQ (Chikungunya Vaccine, Live)
Sponsor: Valneva Austria GmbH
Date of BT Designation Disclosure: 7/6/2021
Approval Date: 11/9/2023
Indication: For the prevention of disease caused by chikungunya virus (CHIKV) in individuals 18 years of age and older.
Category: Infectious Disease
Trade Name (Agent): BAY 2927088
Sponsor: Bayer
Date of BT Designation Disclosure: 2/26/2024
Indication: Treatment for patients with unresectable or metastatic non–small cell lung cancer (NSCLC) whose tumors have activating HER2 mutations, and who have received prior systemic therapy
Category:
Trade Name (Agent): NVL-520
Sponsor: Nuvalent
Date of BT Designation Disclosure: 2/27/2024
Indication: Treatment for patients with ROS1-positive metastatic non-small cell lung cancer (NSCLC) who have been previously treated with two or more ROS1 tyrosine kinase inhibitors (TKIs).
Category: Cancer
Trade Name (Agent): TAGRISSO (osimertinib) + platinum-based chemotherapy
Sponsor: AstraZeneca
Date of BT Designation Disclosure: 8/2023
Approval Date: 2/16/2024
Indication: Treatment for patients with locally advanced or metastatic non–small cell lung cancer (NSCLC) harboring EGFR exon 19 deletions or exon 21 L858R mutations, as detected by an FDA-approved test.
Category: Cancer
Trade Name (Agent): AlphaMedixTM (212Pb-DOTAMTATE)
Sponsor: RadioMedix
Date of BT Designation Disclosure: 1/12/2024
Indication: Treatment of adult patients with unresectable or metastatic, progressive somatostatin receptor (SSTR)–expressing gastroenteropancreatic neuroendocrine tumors (GEP-NETs) who have not previously received peptide receptor radioligand therapy (PRRT).
Category: Cancer
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
