The Breakthrough Therapies chart is an interactive database of all publicly announced Breakthrough Therapy Designations (BTDs) since the program’s inception in 2012. As of March 2026, the U.S. Food and Drug Administration (FDA) reported it has received 1622 BTD requests, granted 634 BTDs, and approved 374 Breakthrough Therapy designated products.
The chart is updated regularly and includes information about each designation including the drug name or investigational code name, the drug sponsor, the date of the designation’s public disclosure, the indication granted BTD, and the current status of the designation.
Click here to learn more about the history of Breakthrough Therapies.
Trade Name (Agent)
Sponsor
Date of BT Designation Disclosure
Approval Date
Indication
Category
Trade Name (Agent): 125I-evuzamitide; AT-01
Sponsor: Attralus, Inc.
Date of BT Designation Disclosure: 8/5/2024
Indication: Diagnostic imaging agent for positron emission tomography (PET) imaging in patients with suspected or known cardiac amyloidosis
Category: Other
Trade Name (Agent): 212Pb-dotamate; 212Pb-DOTAMTATE, SAR447873
Sponsor: RadioMedix and Orano Med
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): ABECMA (idecabtagene vicleucel); bb2121; ide-cel
Sponsor: Bristol Myers Squibb/Bluebird Bio
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): ABRYSVO (respiratory syncytial virus vaccine); RSVpreF; PF-06482077
Sponsor: Pfizer
Date of BT Designation Disclosure: 3/2/2022
Approval Date: 8/21/2023
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): ABRYSVO (respiratory syncytial virus vaccine); RSVpreF; PF-06928316
Sponsor: Pfizer
Date of BT Designation Disclosure: 3/24/2022
Approval Date: 5/31/2023
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): ACTEMRA (tocilizumab); BAT1806
Sponsor: GENENTECH INC
Date of BT Designation Disclosure: 6/10/2015
Approval Date: 3/4/2021
Indication: Systemic scerlosis
Category: Cardio/pulmonary
Trade Name (Agent): ACTEMRA (tocilizumab); BAT1806
Sponsor: GENENTECH INC
Date of BT Designation Disclosure: 10/5/2016
Approval Date: 5/22/2017
Indication: Adult patients with giant cell arteritis (GCA)
Category: Rare Inherited Disorders
Trade Name (Agent): ADAKVEO (crizanlizumab-tmca); SEG101
Sponsor: Novartis
Date of BT Designation Disclosure: 1/8/2019
Approval Date: 11/15/2019
Indication: To reduce the frequency of vasoocclusive crises in adults and pediatric patients aged 16 years and older with sickle cell disease
Category: Rare Inherited Disorders
Trade Name (Agent): ADCETRIS (brentuximab vedotin (+ chemo)); SGN-35
Sponsor: SEATTLE GENETICS INC
Date of BT Designation Disclosure: 10/15/2017
Approval Date: 3/20/2018
Indication: Treatment for adult patients with previously untreated Stage III or IV classical Hodgkin Lymphoma in combination with chemotherapy
Category: Cancer
Trade Name (Agent): ADCETRIS (brentuximab vedotin (+ cyclophosphamide, doxorubicin, and prednisone)); SGN-35
Sponsor: SEATTLE GENETICS INC
Date of BT Designation Disclosure: 11/15/2018
Approval Date: 11/16/2018
Indication: Adult patients with previously untreated systemic anaplastic large cell lymphoma or other CD30-expressing peripheral T-cell lymphomas (PTCL) in combination with cyclophosphamide, doxorubicin, and prednisone
Category: Cancer
Trade Name (Agent): ADCETRIS (brentuximab vedotin); SGN-35
Sponsor: SEATTLE GENETICS INC
Date of BT Designation Disclosure: 10/2/2017
Approval Date: 11/9/2017
Indication: Patients with primary cutaneous anaplastic large cell lymphoma or CD30-expressing mycosis fungoides who have received prior systemic therapy
Category: Cancer
Trade Name (Agent): admilparant; BMS-986278
Sponsor: Bristol-Myers Squibb
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: Cardio/pulmonary
Trade Name (Agent): adrabetadex; VTS-270
Sponsor: Mandos
Date of BT Designation Disclosure: 1/6/2016
Indication: Niemann-Pick Type C1 Disease (NPC)
Category: Rare Inherited Disorders
Trade Name (Agent): ADSTILADRIN (nadofaragene firadenovec-vncg); rAd-IFN/Syn3
Sponsor: Ferring Pharmaceuticals, Inc.
Date of BT Designation Disclosure: 5/9/2018
Approval Date: 12/16/2022
Indication: Treatment of high-grade, Bacillus Calmette-Guérin (BCG) unresponsive, non-muscle invasive bladder cancer (NMIBC)
Category: Cancer
Trade Name (Agent): AFX3772 (formerly ASP3772); GSK-5101955
Sponsor: GlaxoSmithKline
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): ALECENSA (alectinib); CH5424802; AF802
Sponsor: Hoffmann La Roche Inc.
Date of BT Designation Disclosure: 6/15/2013
Approval Date: 12/11/2015
Indication: Patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib
Category: Cancer
Trade Name (Agent): ALECENSA (alectinib); CH5424802; AF802
Sponsor: Hoffmann La Roche Inc.
Date of BT Designation Disclosure: 9/23/2013
Approval Date: 11/6/2017
Indication: Adult patients with advanced ALK-positive non-small cell lung cancer (NSCLC) who have not received prior treatment with an ALK inhibitor
Category: Cancer
Trade Name (Agent): ALIQOPA (copanlisib); BAY 80-6946
Sponsor: Bayer
Date of BT Designation Disclosure: 5/30/2019
Indication: Adults patients with relapsed marginal zone lymphoma (MZL) who have received at least 2 prior therapies
Category: Cancer
Trade Name (Agent): ALTUVIIIO (efanesoctocog alfa); BIVV001
Sponsor: Sanofi & Sobi
Date of BT Designation Disclosure: 5/13/2022
Approval Date: 2/21/2023
Indication: Treatment of people with hemophilia A.
Category: Hematology
Trade Name (Agent): ALUNBRIG (brigatinib); AP-26113
Sponsor: Takeda
Date of BT Designation Disclosure: 10/1/2014
Approval Date: 4/28/2017
Indication: For the treatment of patients with ALK-positive NSCLC whose tumors are resistant to crizotinib.
Category: Cancer
Trade Name (Agent): ANDEXXA (andexanet alfa; coagulation factor xa (recombinant), inactivated-zhzo); PRT-4445
Sponsor: Portola Pharmaceuticals, Inc.
Date of BT Designation Disclosure: 11/25/2013
Approval Date: 5/3/2018
Indication: Patients treated with rivaroxaban and apixaban when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding
Category: Cardio/pulmonary
Trade Name (Agent): ANKTIVA (nogapendekin alfa inbakicept-pmln); N-803
Sponsor: Altor Bioscience LLC/Immunitybio
Date of BT Designation Disclosure: 12/1/2019
Approval Date: 4/22/2024
Indication: In combination with BCG for the treatment of patients with BCG unresponsive non muscle invasive bladder cancer with carcinoma in situ.
Category: Cancer
Trade Name (Agent): APRETUDE (cabotegravir); GSK-1265744; GSK-744
Sponsor: Viiv Healthcare Co.
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): ARCALYST (rilonacept); KPL914
Sponsor: Kiniksa Pharmaceuticals UK
Date of BT Designation Disclosure: 11/20/2019
Approval Date: 3/18/2021
Indication: Treatment of recurrent pericarditis
Category: Cardio/pulmonary
Trade Name (Agent): ARIKAYCE (amikacin liposome inhalation suspension); BAY 41-6551; MAT2501
Sponsor: Insmed Inc.
Date of BT Designation Disclosure: 6/16/2014
Approval Date: 9/28/2018
Indication: Adult patients with nontuberculous mycobacterial lung disease caused by Mycobacterium avium complex.
Category: Infectious Disease
Trade Name (Agent): ARTESUNATE (artesunate); NSC-712571
Sponsor: Amivas
Date of BT Designation Disclosure: 9/11/2018
Approval Date: 5/26/2020
Indication: Severe malaria
Category: Infectious Disease
Trade Name (Agent): ARZERRA (ofatumumab); GSK-1841157
Sponsor: Novartis
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): atacicept; TACI-Ig; TACI-Fc 5
Sponsor: Vera Therapeutics, Inc.
Date of BT Designation Disclosure: 5/28/2024
Indication: Treatment of IgA nephropathy (IgAN).
Category: Rare Inherited Disorders
Trade Name (Agent): AUGTYRO (repotrectinib); TPX-005; BMS-986472
Sponsor: Bristol-Myers Squibb
Date of BT Designation Disclosure: 12/7/2020
Approval Date: 11/15/2023
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-naive).
Category: Cancer
Trade Name (Agent): AUGTYRO (repotrectinib); TPX-005; BMS-986472
Sponsor: Bristol-Myers Squibb
Date of BT Designation Disclosure: 10/4/2021
Approval Date: 6/13/2024
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 options.
Category: Cancer
Trade Name (Agent): AUGTYRO (repotrectinib); TPX-005; BMS-986472
Sponsor: Bristol-Myers Squibb
Date of BT Designation Disclosure: 5/9/2022
Approval Date: 11/15/2023
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): AUSTEDO (deutetrabenazine); SD-809
Sponsor: Teva Pharmaceuticals
Date of BT Designation Disclosure: 11/9/2015
Approval Date: 8/30/2017
Indication: Patients with tardive dyskinesia
Category: Neuro/Psych
Trade Name (Agent): AUVELITY (dextromethorphan-bupropion); AXS-05
Sponsor: Axsome Therapeutics, Inc.
Date of BT Designation Disclosure: 3/25/2019
Approval Date: 8/18/2022
Indication: Major Depressive Disorder (MDD)
Category: Neuro/Psych
Trade Name (Agent): AUVELITY (dextromethorphan-bupropion); AXS-05
Sponsor: Axsome Therapeutics
Date of BT Designation Disclosure: 6/26/2020
Indication: Treatment of Alzheimer's disease agitation
Category: Neuro/Psych
Trade Name (Agent): avasopasem; GC4419
Sponsor: Galera Therapeutics
Date of BT Designation Disclosure: 2/28/2018
Indication: Reduction of the duration severity and incidence of severe oral mucositis induced by radiation therapy
Category: Other
Trade Name (Agent): AVASTIN (bevacizumab (+ atezolizumab)); ABP-215
Sponsor: GENENTECH INC
Date of BT Designation Disclosure: 7/13/2018
Approval Date: 5/29/2020
Indication: In combination with atezolizumab for the first-line treatment of patients with advanced or metastatic HCC.
Category: Cancer
Trade Name (Agent): avexitide; EXENDIN 9-39
Sponsor: Eiger (now under Amylyx)
Date of BT Designation Disclosure: 6/17/2019
Indication: Post-bariatric hypoglycemia (PBH)
Category: Other
Trade Name (Agent): avexitide; EXENDIN 9-39
Sponsor: Eiger (now under Amylyx)
Date of BT Designation Disclosure: 8/5/2021
Indication: Treatment of congenital hyperinsulism (HI).
Category: Other
Trade Name (Agent): AVMAPKI FAKZYNJA CO-PACK (avutometinib + defactinib); VS-6766 + VS-6063
Sponsor: Verastem
Date of BT Designation Disclosure: 5/24/2021
Approval Date: 5/8/2025
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): AYVAKIT (avapritinib); BLU-285;70C366;X-720776
Sponsor: Blueprint Medicines Corp
Date of BT Designation Disclosure: 10/12/2018
Approval Date: 6/16/2021
Indication: For advanced systemic mastocytosis.
Category: Cancer
Trade Name (Agent): AYVAKIT (avapritinib); BLU-285;70C366;X-720776
Sponsor: Blueprint Medicines Corp
Date of BT Designation Disclosure: 12/23/2020
Approval Date: 5/22/2023
Indication: For treatment of moderate to severe indolent systemic mastocytosis (ISM).
Category: Cancer
Trade Name (Agent): AYVAKIT (avapritinib); BLU-285;70C366;X-720776
Sponsor: Blueprint Medicines Corp
Date of BT Designation Disclosure: 6/1/2017
Approval Date: 1/9/2020
Indication: Treatment of patients with unresectable or metastatic GIST harboring the PDGFRα D842V mutation.
Category: Cancer
Trade Name (Agent): AZEDRA (iobenguane I 131); iobenguane I 131
Sponsor: Progenics Pharmaceuticals Inc.
Date of BT Designation Disclosure: 7/26/2015
Approval Date: 7/30/2018
Indication: For the treatment of patients with iobenguane-avid metastatic and/or recurrent pheochromocytoma and paraganglioma (PPGL)
Category: Cancer
Trade Name (Agent): balovaptan; RG7314
Sponsor: Roche
Date of BT Designation Disclosure: 1/28/2018
Indication: A vasopressin 1a (V1a) receptor antagonist for patients with autism spectrum disorder (ASD)
Category: Neuro/Psych
Trade Name (Agent): BALVERSA (erdafitinib); JNJ-42756493
Sponsor: Janssen Biotech Inc.
Date of BT Designation Disclosure: 3/13/2018
Approval Date: 4/12/2019
Indication: For the treatment of adult patients with locally advanced or metastatic urothelial carcinoma.
Category: Cancer
Trade Name (Agent): BAVENCIO (avelumab (+ axitinib)); MSB0010718C
Sponsor: EMD Serono Inc.
Date of BT Designation Disclosure: 12/21/2017
Approval Date: 5/14/2019
Indication: In combination with Inlyta (axitinib) for first-line treatment of patients with advanced renal cell carcinoma (RCC)
Category: Cancer
Trade Name (Agent): BAVENCIO (avelumab); MSB0010718C
Sponsor: EMD Serono Inc.
Date of BT Designation Disclosure: 11/17/2015
Approval Date: 3/23/2017
Indication: For the treatment of patients with metastatic Merkel cell carcinoma (mMCC).
Category: Cancer
Trade Name (Agent): BAVENCIO (avelumab); MSB0010718C
Sponsor: EMD Serono Inc.
Date of BT Designation Disclosure: 3/26/2020
Approval Date: 6/30/2020
Indication: Maintenance treatment for patients with urothelial carcinoma (mUC).
Category: Cancer
Trade Name (Agent): bemarituzumab (+ modified FOLFOX chemo); FPA-144
Sponsor: Amgen (Five Prim Therapeutics)
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 modified FOLFOX6 chemotherapy
Category: Cancer
Trade Name (Agent): bempegaldesleukin + nivolumab; NKTR-214
Sponsor: Nektar Therapeutics
Date of BT Designation Disclosure: 8/23/2019
Indication: Untreated melanoma patients with either metastatic disease or who are unable to undergo surgery
Category: Cancer
Trade Name (Agent): BENLYSTA (belimumab); GSK1550188
Sponsor: GlaxoSmithKline LLC
Date of BT Designation Disclosure: 4/29/2020
Approval Date: 12/16/2020
Indication: Lupus nephritis
Category: Other
Trade Name (Agent): bentracimab; PB2452
Sponsor: SFJ Pharmaceuticals and PhaseBio Pharmaceuticals (now SERB Pharmaceuticals)
Date of BT Designation Disclosure: 4/8/2019
Indication: Reversal agent for the antiplatelet drug ticagrelor
Category: Hematology
Trade Name (Agent): BEQVEZ (fidanacogene elaparvovec-dzkt); SPK-9001; PF-06838435
Sponsor: Pfizer Inc.
Date of BT Designation Disclosure: 7/15/2016
Approval Date: 4/25/2024
Indication: Treatment of Hemophilia B.
Category: Rare Inherited Disorders
Trade Name (Agent): BESPONSA (inotuzumab ozogamicin); CMC-544
Sponsor: Wyeth Pharmaceuticals LLC
Date of BT Designation Disclosure: 10/15/2015
Approval Date: 8/17/2017
Indication: For the treatment of relapsed or refractory B-cell ALL.
Category: Cancer
Trade Name (Agent): bexicaserin; LP352
Sponsor: Longboard Pharmaceuticals, Inc.
Date of BT Designation Disclosure: 7/1/2024
Indication: For the treatment of seizures associated with Developmental and Epileptic Encephalopathies (DEEs)
Category: Neuro/Psych
Trade Name (Agent): BEXSERO (Meningococcal Group B Vaccine); rMenB+OMV
Sponsor: GlaxoSmithKline Biologicals
Date of BT Designation Disclosure: 4/1/2014
Approval Date: 1/23/2015
Indication: For use to prevent invasive disease caused by Neisseria meningitidis serogroup B in individuals 10 through 25 years of age.
Category: Infectious Disease
Trade Name (Agent): BEYFORTUS (nirsevimab-alip); MEDI8897
Sponsor: AstraZeneca (formerly MedImmune)
Date of BT Designation Disclosure: 12/3/2018
Approval Date: 7/17/2023
Indication: For the prevention of RSV lower respiratory tract disease healthy preterm infants entering their first RSV season, and children up to 24 months of age with CLD or CHD.
Category: Infectious Disease
Trade Name (Agent): BI 6727 (volasertib (combination therapy))
Sponsor: Boehringer Ingelheim Pharmaceuticals Inc
Date of BT Designation Disclosure: 9/17/2013
Indication: AML combination therapy
Category: Cancer
Trade Name (Agent): bimagrumab; BYM338
Sponsor: Novartis Pharmaceuticals Corp.
Date of BT Designation Disclosure: 8/20/2013
Indication: Sporadic inclusion body myositis (sIBM)
Category: Neuro/Psych
Trade Name (Agent): BIZENGRI (zenocutuzumab); MCLA-128
Sponsor: Merus Nv
Date of BT Designation Disclosure: 7/3/2023
Approval Date: 12/4/2024
Indication: For NRG1+ NSCLC.
Category: Cancer
Trade Name (Agent): BIZENGRI (zenocutuzumab); MCLA-128
Sponsor: Merus Nv
Date of BT Designation Disclosure: 5/17/2023
Approval Date: 12/4/2024
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): BLENREP (belantamab mafodoton-blmf); GSK 2857916
Sponsor: GlaxoSmithKline Intellectual Property Development LTD England
Date of BT Designation Disclosure: 10/31/2017
Approval Date: 8/5/2020
Indication: For multiple myeloma patients who have failed at least 3 prior lines of therapy including an anti-CD38 antibody and are refractory to a proteasome inhibitor and an immunomodulatory agent.
Category: Cancer
Trade Name (Agent): BLINCYTO (blinatumomab); AMG103; MT103
Sponsor: Amgen Inc.
Date of BT Designation Disclosure: 6/30/2014
Approval Date: 12/3/2014
Indication: Philadelphia chromosome-negative relapsed or refractory B-precursor acute lymphoblastic leukemia (ALL).
Category: Cancer
Trade Name (Agent): BLINCYTO (blinatumomab); AMG103; MT103
Sponsor: Amgen Inc.
Date of BT Designation Disclosure: 9/30/2023
Approval Date: 6/14/2024
Indication: CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (ALL).
Category: Cancer
Trade Name (Agent): BRAFTOVI + MEKTOVI + ERBITUX (encorafenib + binimetinib + cetuximab); LGX818
Sponsor: Array BioPharma Inc.
Date of BT Designation Disclosure: 8/7/2018
Approval Date: 4/8/2020
Indication: In combination with cetuximab, for the treatment of adult patients with metastatic colorectal cancer (CRC) with a BRAF V600E mutation, as detected by an FDA-approved test, after prior therapy
Category: Cancer
Trade Name (Agent): BREYANZI (lisocabtagene maraleucel); JCAR017
Sponsor: Juno Therapeutics, A Celgene Company
Date of BT Designation Disclosure: 12/15/2016
Approval Date: 2/5/2021
Indication: For treatment of subjects with relapsed/refractory large B-cell lymphoma (including Diffuse Large B-cell Lymphoma not otherwise specified-de novo or transformed from indolent lymphoma, PMBCL and grade 3B Follicular Lymphoma).
Category: Cancer
Trade Name (Agent): BRINEURA (cerliponase alfa); BMN-190
Sponsor: BioMarin Pharmaceutical
Date of BT Designation Disclosure: 8/27/2016
Approval Date: 4/27/2017
Indication: Pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) also known as tripeptidyl peptidase 1 (TPP1) deficiency.
Category: Rare Inherited Disorders
Trade Name (Agent): BRINSUPRI (brensocatib); INS1007
Sponsor: Insmed Inc.
Date of BT Designation Disclosure: 6/8/2020
Approval Date: 8/12/2025
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): brogidirsen; 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: Neuro/Psych
Trade Name (Agent): BRUKINSA (zanubrutinib); BGB-3111
Sponsor: BeiGene USA, Inc.
Date of BT Designation Disclosure: 1/9/2019
Approval Date: 11/14/2019
Indication: For the treatment of patients with mantle cell lymphoma (MCL) who have received at least one prior therapy.
Category: Cancer
Trade Name (Agent): bulevirtide; Formerly myrcludex; approved in EU as HEPCLUDEX
Sponsor: Gilead Sciences Inc.
Date of BT Designation Disclosure: 10/23/2018
Indication: Chronic hepatitis delta (HDV) infection
Category: Infectious Disease
Trade Name (Agent): CABOMETYX (cabozantinib); XL184
Sponsor: Exelixis Inc.
Date of BT Designation Disclosure: 8/21/2015
Approval Date: 4/25/2016
Indication: Patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.
Category: Cancer
Trade Name (Agent): CABOMETYX (cabozantinib); XL184
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): cadisegliatin; TTP399
Sponsor: vTv Therapeutics
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): CALQUENCE (acalabrutinib); ACP-196
Sponsor: AstraZeneca UK Ltd
Date of BT Designation Disclosure: 7/31/2017
Approval Date: 10/31/2017
Indication: For the treatment of patients with mantle cell lymphoma (MCL) who have received at least one prior therapy.
Category: Cancer
Trade Name (Agent): CALQUENCE (acalabrutinib); ACP-196
Sponsor: AstraZeneca UK Ltd
Date of BT Designation Disclosure: 8/12/2019
Approval Date: 11/21/2019
Indication: Monotherapy for treatment of adult patients with CLL
Category: Cancer
Trade Name (Agent): CAMZYOS (mavacamten); MYK-461
Sponsor: Myokardia Inc
Date of BT Designation Disclosure: 4/27/2016
Approval Date: 4/28/2022
Indication: Treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in adults to improve functional capacity.
Category: Rare Inherited Disorders
Trade Name (Agent): CAMZYOS (mavacamten); MYK-461
Sponsor: Myokardia Inc
Date of BT Designation Disclosure: 7/22/2020
Approval Date: 4/28/2022
Indication: Treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in adults to improve symptoms.
Category: Rare Inherited Disorders
Trade Name (Agent): CAPVAXIVE (pneumococcal 21-valent conjugate vaccine); V116
Sponsor: Merck
Date of BT Designation Disclosure: 4/14/2022
Approval Date: 6/17/2024
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): CARVYKTI (ciltacabtagene autoleucel); JNJ-4528; LCAR-B38M CAR-T cells LCAR-B38M
Sponsor: Janssen Biotech Inc.
Date of BT Designation Disclosure: 12/6/2019
Approval Date: 2/28/2022
Indication: Patients with previously treated multiple myeloma
Category: Cancer
Trade Name (Agent): CIBINQO (abrocitinib); PF-04965842
Sponsor: Pfizer Inc.
Date of BT Designation Disclosure: 2/7/2018
Approval Date: 1/14/2022
Indication: Patients with moderate-to-severe atopic dermatitis (AD)
Category: Other
Trade Name (Agent): CMS I-NEB (colistimethate sodium powder for nebulization solution); 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: Cardio/pulmonary
Trade Name (Agent): COSELA (trilaciclib); G1T28
Sponsor: G1 Therapeutics Inc
Date of BT Designation Disclosure: 8/1/2019
Approval Date: 2/12/2021
Indication: Reduce the frequency of chemotherapy-induced bone marrow suppression in adults receiving certain types of chemotherapy for extensive-stage (when the cancer has spread beyond the lungs) small cell lung cancer.
Category: Cancer
Trade Name (Agent): COTELLIC (cobimetinib); GDC 0973;XL 518;RG 7420;RO5514041;XL 518
Sponsor: Genentech/Roche
Date of BT Designation Disclosure: 10/3/2019
Indication: Adult patients with histiocytic neoplasms who do not harbor a BRAF V600 mutation
Category: Rare Inherited Disorders
Trade Name (Agent): CP101
Sponsor: Finch Therapeutics
Date of BT Designation Disclosure: 2/9/2019
Indication: Patients with recurrent Clostridium difficile infection
Category: Infectious Disease
Trade Name (Agent): CRENESSITY (crinecerfont); NBI-74788
Sponsor: Neurocrine Biosciences Inc.
Date of BT Designation Disclosure: 12/1/2023
Approval Date: 12/13/2024
Indication: Treatment of congenital adrenal hyperplasia
Category: Other
Trade Name (Agent): cretostimogene grenadenorepvec; CG0070
Sponsor: CG Oncology
Date of BT Designation Disclosure: 12/5/2023
Indication: Immunotherapy for treatment of high-risk 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): CRS-207 + GVAX
Sponsor: Aduro
Date of BT Designation Disclosure: 7/21/2014
Indication: Pancreatic cancer
Category: Cancer
Trade Name (Agent): CRYSVITA (burosumab); KRN-23
Sponsor: Kyowa Kirin Inc.
Date of BT Designation Disclosure: 6/22/2016
Approval Date: 4/17/2018
Indication: For the treatment of XLH in pediatric patients one year of age and older.
Category: Rare Inherited Disorders
Trade Name (Agent): cytisinicline (cytisine); cytisinicline (cytisine)
Sponsor: Achieve Life Sciences
Date of BT Designation Disclosure: 7/31/2024
Indication: For nicotine e-cigarette cessation
Category: Other
Trade Name (Agent): D-cycloserine/lurasidone; NRX-101
Sponsor: NRx Pharmaceuticals
Date of BT Designation Disclosure: 11/13/2018
Indication: For treatment of Severe Bipolar Depression with Acute Suicidal Ideation & Behavior (ASIB) after initial stabilization with ketamine or other effective therapy
Category: Neuro/Psych
Trade Name (Agent): DAKLINZA (daclatasvir + asunaprevir); BMS 790052
Sponsor: Bristol-Myers Squibb Company
Date of BT Designation Disclosure: 2/3/2014
Approval Date: 2/5/2016
Indication: DCV/ASV (DUAL) combination therapy for the treatment of genotype 1b, chronic hepatitis C virus infection
Category: Infectious Disease
Trade Name (Agent): DANYELZA (naxitamab-gqgk); humanized 3F8 monoclonal antibody (hu3F8)
Sponsor: Y-mAbs Therapeutics, Inc.
Date of BT Designation Disclosure: 8/20/2018
Approval Date: 11/25/2020
Indication: Naxitamab in combination with GM-CSF, for the treatment of high-risk neuroblastoma refractory to initial therapy or with incomplete response to salvage therapy in patients > 12 months of age with persistent, refractory disease limited to bone marrow with or without evidence of concurrent bone involvement.
Category: Cancer
Trade Name (Agent): DARZALEX (daratumumab); JNJ-54767414
Sponsor: Janssen Biotech Inc.
Date of BT Designation Disclosure: 5/1/2013
Approval Date: 11/16/2015
Indication: Patients with multiple myeloma who have received at least 3 prior lines of therapy including a proteasome inhibitor and an immunomodulatory agent or are double refractory to a proteasome inhibitor and an immunomodulatory agent.
Category: Cancer
Trade Name (Agent): DARZALEX (daratumumab); JNJ-54767414
Sponsor: Janssen Biotech Inc.
Date of BT Designation Disclosure: 7/26/2016
Approval Date: 11/21/2016
Indication: Patients with multiple myeloma who have received at least one prior therapy in combination with lenalidomide and dexamethasone OR in combination with bortezomib and dexamethasone.
Category: Cancer
Trade Name (Agent): DATROWAY (datopotamab deruxtecan); Dato-DXd; DS-1062a
Sponsor: AstraZeneca & Daiichi Sankyo
Date of BT Designation Disclosure: 12/9/2024
Approval Date: 6/23/2025
Indication: For the treatment of adult patients with locally advanced or metastatic epidermal growth factor receptor-mutated (EGFR-mutated) non-small cell lung cancer (NSCLC) who have received prior systemic therapies, including an EGFR-directed therapy.
Category: Cancer
Trade Name (Agent): delpacibart etedesiran; AOC 1001; del-desiran
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): delpacibart zotadirsen; AOC-1044; del-zota
Sponsor: Avidity
Date of BT Designation Disclosure: 7/23/2025
Indication: Treatment of Duchenne muscular dystrophy amenable to exon 44 skipping (DMD44)
Category: Neuro/Psych
Trade Name (Agent): denifanstat; TVB-2640; 1399177-37-7; FASN-IN-2
Sponsor: Sagimet Biosciences Inc.
Date of BT Designation Disclosure: 10/1/2024
Indication: For treatment of noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH) with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis).
Category: Other
Trade Name (Agent): deuterated psilocin; CYB003
Sponsor: Cybin, Inc
Date of BT Designation Disclosure: 3/14/2024
Indication: Adjunctive treatment for major depressive disorder (MDD)
Category: Neuro/Psych
