List of drugs granted breakthrough therapy designation
March 16, 2024
Drugs granted breakthrough therapy designation (BTD) by the US Food and Drug Administration (FDA). Drugs may be listed more than once since breakthrough therapy can be awarded for multiple indications.
^ abcdefghiNew Drug Therapy Approvals 2023 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2024. from the original on 10 January 2024. Retrieved 9 January 2024. This article incorporates text from this source, which is in the public domain.
^ ab"Breakthrough Therapy Approvals". U.S. Food and Drug Administration (FDA). 2020-10-08. from the original on 2023-06-11. Retrieved 2023-06-25.
^"Approval report" (PDF). U.S. Food and Drug Administration (FDA). (PDF) from the original on 2019-04-24. Retrieved 2019-06-12.
March 16, 2024
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Drugs granted breakthrough therapy designation BTD by the US Food and Drug Administration FDA Drugs may be listed more than once since breakthrough therapy can be awarded for multiple indications Contents 1 2023 2 2022 3 2021 4 2020 5 2019 6 2018 7 2017 8 2016 9 2015 10 2014 11 2013 12 References2023 editBrand name Manufacturer IndicationElrexfio To treat relapsed or refractory multiple myeloma after at least four lines of therapy 1 Fabhalta To treat paroxysmal nocturnal hemoglobinuria 1 Izervay To treat geographic atrophy secondary to age related macular degeneration 1 Leqembi To treat Alzheimer s disease 1 Loqtorzi To treat recurrent or metastatic nasopharyngeal carcinoma with or following other therapies 1 Ogsiveo To treat desmoid tumors 1 Pombiliti To treat late onset Pompe disease with miglustat 1 Rivfloza To lower urinary oxalate levels in primary hyperoxaluria type 1 and relatively preserved kidney function 1 Talvey To treat relapsed or refractory multiple myeloma after at least four therapies 1 2022 editDrug Manufacturer IndicationAbrocitinib Pfizer atopic dermatitisTebentafusp Immunocore HLA A 02 01 positive uveal melanomaSutimlimab Bioverativ cold agglutinin diseaseLutetium 177Lu vipivotide tetraxetan Novartis prostate specific membrane antigen PSMA positive metastatic castration resistant prostate cancer mCRPC Alpelisib Novartis PIK3CA related overgrowth spectrum conditionsMavacamten Myokardia Inc class II III obstructive hypertrophic cardiomyopathyTrastuzumab deruxtecan Daiichi Sankyo granted for three indications HER2 positive breast cancer with prior anti HER2 based treatment HER2 low IHC 1 or IHC 2 ISH breast cancer and non small cell lung cancer with an activating HER2 mutationDupilumab Regeneron Pharmaceuticals granted for two indications eosinophilic esophagitis and atopic dermatitisIvosidenib Servier Pharmaceuticals acute myeloid leukemia with a susceptible IDH1 mutationBaricitinib Eli Lilly and Co severe alopecia areataSetmelanotide Rhythm Pharmaceuticals Bardet Biedl syndromeDextromethorphan bupropion Axsome Therapeutics major depressive disorderPemigatinib Incyte myeloid lymphoid neoplasms with FGFR1 rearrangementOlipudase alfa Genzyme non central nervous system manifestations of acid sphingomyelinase deficiencySpesolimab Boehringer Ingelheim generalized pustular psoriasisFutibatinib Taiho Oncology intrahepatic cholangiocarcinoma with FGFR2 gene fusions or other rearrangementsTeclistamab Janssen multiple myelomaTeplizumab Provention Bio delay the onset of Stage 3 type 1 diabetes T1D in Stage 2 T1DAtezolizumab Genentech alveolar soft part sarcomaAdagrasib Mirati Therapeutics KRAS G12C mutated non small cell lung cancerMosunetuzumab Genentech follicular lymphomaLenacapavir Gilead Sciences HIV 12021 editDrug Manufacturer IndicationCrizotinib PF Prism ALK positive systemic anaplastic large cell lymphomaTrastuzumab deruxtecan Daiichi Sankyo HER2 positive gastric or gastroesophageal junction adenocarcinoma previously treated with trastuzumabUmbralisib TG Therapeutics marginal zone lymphoma previously treated with an anti CD20 based regimenEvinacumab Regeneron Pharmaceuticals homozygous familial hypercholesterolemiaTrilaciclib G1 Therapeutics myelosuppression when administered prior to a platinum etoposide or topotecan containing regimen for small cell lung cancerFosdenopterin Origin Biosciences molybdenum cofactor deficiency Type ATocilizumab Genentech systemic sclerosis associated interstitial lung diseaseRilonacept Kiniksa Pharmaceuticals recurrent pericarditisDostarlimab GSK mismatch repair deficient recurrent or advanced endometrial cancerDapagliflozin AstraZeneca chronic kidney diseaseBupivacaine meloxicam Heron Therapeutics postsurgical analgesiaAmivantamab Janssen Biotech non small cell lung cancer with EGFR exon 20 insertion mutationsSotorasib Amgen non small cell lung cancer with KRAS G12C mutationAvapritinib Blueprint Medicines Corp granted for two indications mast cell leukemia and advanced systemic mastocytosisBelumosudil Kadmon Pharmaceuticals chronic graft versus host diseasePembrolizumab Merck high risk early stage triple negative breast cancerAvalglucosidase alfa Genzyme late onset Pompe diseaseLenvatinib Eisai Inc advanced renal cell carcinoma used in combination with pembrolizumabDifelikefalin Cara Therapeutics pruritis related to hemodialysis in chronic kidney diseaseMobocertinib Takeda Pharmaceuticals non small cell lung cancer with EGFR exon 20 insertion mutationsCabozantinib Exelixis thyroid cancerMaralixibat Mirum Pharmaceuticals cholestatic pruritus in Alagille syndromeAsciminib Novartis granted for two indications Philadelphia chromosome positive chronic myeloid leukemia previously treated with tyrosine kinase inhibitors and Philadelphia chromosome positive chronic myeloid leukemia with T315I mutationSirolimus AADI Bioscience metastatic or unresectable perivascular epithelioid cell tumorMaribavir Takeda refractory post transplant cytomegalovirus infectionAbatacept Bristol Myers Squibb acute graft versus host disease in combination with a calcineurin inhibitor and methotrexate undergoing hematopoietic stem cell transplantation from a matched or 1 allele mismatched unrelated donorVoxelotor Global Blood Therapeutics Inc sickle cell diseaseCabotegravir ViiV Healthcare Co pre exposure prophylaxis for HIV2020 editDrug Manufacturer IndicationAvapritinib Blueprint Medicines Corp metastatic gastrointestinal stromal tumorTeprotumumab trbw Horizon Therapeutics thyroid eye diseaseNintedanib Boehringer Ingelheim chronic fibrosing interstitial lung diseasesIpilimumab Bristol Myers Squibb hepatocellular carcinomaNivolumab Bristol Myers Squibb hepatocellular carcinomaSelumetinib AstraZeneca neurofibromatosis type 1Mitomycin Urogen Pharma upper tract urothelial cancerTucatinib Seattle Genetics advanced unresectable or metastatic HER2 positive breast cancerPemigatinib Incyte Corp metastatic cholangiocarcinoma with FGFR2 fusionSacituzumab govitecan hziy Immunomedics metastatic triple negative breast cancerCapmatinib Novartis metastatic non small cell lung cancerSelpercatinib Loxo Oncology metastatic RET fusion positive non small cell lung cancerPomalidomide Celgene Corp AIDS related Kaposi sarcomaRucaparib Clovis Oncology metastatic castration resistant prostate cancerRipretinib Deciphera Pharmaceuticals advanced gastrointestinal stromal tumorArtesunate Amivas LLC severe malariaBevacizumab Genentech metastatic hepatocellular carcinomaAtezolizumab Genentech metastatic hepatocellular carcinomaInebilizumab cdon Viela Bio neuromyelitis optica spectrum disorderFostemsavir Viiv Healthcare multidrug resistant HIV 1Tafasitamab cxix MorphoSys relapsed or refractory diffuse large B cell lymphomaEsketamine Janssen major depressive disorder with acute suicidal ideation or behavior 2 belantamab mafodotin blmf GlaxoSmithKline refractory multiple myelomaSatralizumab mwge Genentech neuromyelitis optica spectrum disorderPralsetinib Blueprint Medicines Corp RET fusion positive non small cell lung cancerPitolisant Harmony Biosciences cataplexy in adults with narcolepsy 2 Atoltivimab maftivimab odesivimab ebgn Regeneron Ebola ZaireLonafarnib Eiger BioPharmaceuticals Hutchinson Gilford progeria syndrome HGPS processing deficient progeroid laminopathies with LMNA or ZMPSTE24 mutations 2 Lumasiran Alnylam Pharmaceuticals primary hyperoxaluria type 1 2 Naxitamab gqgk Y mAbs Therapeutics relapsed or refractory high risk neuroblastoma used in combination with GM CSFSetmelanotide Rhythm Pharmaceuticals medical obesity caused by proopiomelanocortin POMC proprotein convertase subtilisin kexin type 1 PCSK1 or leptin receptor LEPR deficiencyPralsetinib Blueprint Medicines Corp RET mutant medullary thyroid cancer or advanced or metastatic RET fusion positive thyroid cancer in radioactive iodine refractory patients 2 Belimumab GlaxoSmithKline active lupus nephritis 2 Osimertinib AstraZeneca adjuvant therapy after tumor resection in non small cell lung cancer with EGFR exon 19 deletions or exon 21 L858R mutationsAnsuvimab zykl Ridgeback Biotherapeutics Ebola Zaire2019 editDrug Manufacturer IndicationLetrozole ribociclib 2 Novartis Pharmaceuticals HR positive HER2 negative advanced or metastatic breast cancer 2 Esketamine Janssen treatment resistant depression 2 Dupilumab Regeneron Pharmaceuticals atopic dermatitis 2 Brexanolone Sage Therapeutics postpartum depression 2 Palbociclib Pfizer metastatic breast cancer 2 Erdafitinib Janssen urothelial carcinoma 2 Pembrolizumab Merck Sharp amp Dohme advanced renal cell carcinoma 2 Ado trastuzumab emtansine Genentech HER2 positive early breast cancer 2 Tafamidis meglumine Foldrx Pharmaceuticals cardiomyopathy 2 Avelumab EMD Serono advanced renal cell carcinoma 2 Venetoclax AbbVie chronic lymphocytic leukemiaor small lymphocytic lymphoma 2 Galcanezumab Eli Lilly episodic cluster headache 2 Polatuzumab vedotin piiq Genentech diffuse large B cell lymphoma 2 Pexidartinib Daiichi Sankyo tenosynovial giant cell tumor 2 Entrectinib Genentech solid tumors that have a neurotrophic tyrosine receptor kinase NTRK gene fusion 2 Pembrolizumab Merck endometrial carcinoma 2 Lenvatinib Eisai endometrial carcinoma 2 Elexacaftor tezacaftor ivacaftor Vertex Pharmaceuticals cystic fibrosis 2 Zanubrutinib BeiGene mantle cell lymphoma 2 Crizanlizumab tmca Novartis sickle cell disease 2 Givosiran Alnylam Pharmaceuticals acute hepatic porphyria 2 Acalabrutinib AstraZeneca relapsed or refractory chronic lymphocytic leukemia 2 Acalabrutinib AstraZeneca untreated chronic lymphocytic leukemia 2 Voxelotor Global Blood Therapeutics sickle cell disease 2 Enfortumab vedotin ejfv Astellas Pharma metastatic urothelial carcinoma 2 Fam trastuzumab deruxtecan nxki Daiichi Sankyo HER2 positive breast cancer 2 3 2018 editDrug Manufacturer IndicationTezacaftor ivacaftor 2 Vertex Pharmaceuticals cystic fibrosisDurvalumab 2 AstraZeneca UK unresectable Stage III non small cell lung cancerIbalizumab uiyk 2 Theratechnologies human immunodeficiency virus type 1 HIV 1 infectionBrentuximab vedotin 2 Seattle Genetics Hodgkin lymphomaIpilimumab 2 Bristol Myers Squibb renal cell carcinomaNivolumab 2 Bristol Myers Squibb renal cell carcinomaBurosumab twza 2 Kyowa Hakko Kirin X linked hypophosphatemiaOsimertinib 2 AstraZeneca metastatic non small cell lung cancerDabrafenib 2 Novartis Pharmaceuticals melanoma with BRAF V600E or V600K mutationsTrametinib 2 Novartis Pharmaceuticals melanoma with BRAF V600E or V600K mutationsDabrafenib 2 Novartis Pharmaceuticals anaplastic thyroid cancer with BRAF V600E mutationTrametinib 2 Novartis Pharmaceuticals anaplastic thyroid cancer with BRAF V600E mutationFingolimod 2 Novartis Pharmaceuticals multiple sclerosisRituximab 2 Genentech pemphigus vulgarisVenetoclax 2 AbbVie chronic lymphocytic leukemiaPembrolizumab 2 Merck Sharp amp Dohme mediastinal large B cell lymphomaIpilimumab 2 Bristol Myers Squibb metastatic colorectal cancerNivolumab 2 Bristol Myers Squibb metastatic colorectal cancerRibociclib 2 Novartis Pharmaceuticals breast cancerTafenoquine 2 GlaxoSmithKline Plasmodium vivax malariaIobenguane I 131 2 Progenics Pharmaceuticals pheochromocytoma or paragangliomaLumacaftor ivacaftor 2 Vertex Pharmaceuticals cystic fibrosisMogamulizumab kpkc 2 Kyowa Hakko Kirin mycosis fungoides or Sezary syndromePatisiran 2 Alnylam Pharmaceuticals hereditary transthyretin mediated amyloidosisCenegermin bkbj 2 Dompe Farmaceutici neurotrophic keratitisLanadelumab flyo 2 Takeda Pharmaceuticals hereditary angioedemaCemiplimab rwlc 2 Regeneron Pharmaceuticals cutaneous squamous cell carcinomaAmikacin liposome inhalation suspension 2 Insmed mycobacterial lung diseaseEmicizumab kxwh 2 Genentech hemophiliaLorlatinib 2 Pfizer ALK positive metastatic non small cell lung cancerBrentuximab vedotin 2 Seattle Genetics anaplastic large cell lymphomaEltrombopag 2 Novartis Pharmaceuticals severe aplastic anemiaEmapalumab lzsg 2 Swedish Orphan Biovitrum hemophagocytic lymphohistiocytosisVenetoclax 2 AbbVie acute myeloid leukemiaLarotrectinib 2 Bayer Healthcare solid tumors that have a neurotrophic receptor tyrosine kinase NTRK gene fusionLarotrectinib 2 Bayer Healthcare solid tumors that have a neurotrophic receptor tyrosine kinase NTRK gene fusionAmifampridine 2 Catalyst Pharmaceuticals Lambert Eaton myasthenic syndromePembrolizumab 2 Merck Sharp amp Dohme Merkel cell carcinomaTagraxofusp erzs 2 Stemline Therapeutics blastic plasmacytoid dendritic cell neoplasm 3 2017 edit 4 Drug Manufacturer IndicationNivolumab 2 Bristol Myers Squibb urothelial carcinomaRibociclib 2 Novartis Pharmaceuticals HR positive HER2 negative breast cancerPembrolizumab 2 Merck Sharp amp Dohme Hodgkin LymphomaAvelumab 2 EMD Serono metastatic Merkel cell carcinomaNiraparib 2 GlaxoSmithKline recurrent ovarian cancer responsive to platinum based chemotherapyOcrelizumab 2 Genentech multiple sclerosisDupilumab 2 Regeneron Pharmaceuticals atopic dermatitisPalbociclib 2 Pfizer HR positive HER2 negative breast cancerValbenazine 2 Neurocrine Biosciences tardive dyskinesiaRanibizumab 2 Genentech diabetic retinopathyCerliponase alfa 2 BioMarin Pharmaceutical tripeptidyl peptidase 1 TPP1 deficiencyMidostaurin 2 Novartis Pharmaceuticals FLT3 positive acute myeloid leukemiaBrigatinib 2 Takeda Pharmaceuticals ALK positive non small cell lung cancerDurvalumab 2 AstraZeneca UK metastatic urothelial carcinomaLetrozole ribociclib 2 Novartis Pharmaceuticals breast cancerPembrolizumab 2 Merck Sharp amp Dohme urothelial carcinomaTocilizumab 2 Genentech giant cell arteritisPembrolizumab 2 Merck Sharp amp Dohme microsatellite instability high tumorsCeritinib 2 Novartis Pharmaceuticals ALK positive metastatic non small cell lung cancerDabrafenib 2 Novartis Pharmaceuticals metastatic non small cell lung cancer with BRAF V600E mutationTrametinib 2 Novartis Pharmaceuticals metastatic non small cell lung cancer with BRAF V600E mutationIbrutinib 2 Pharmacyclics chronic graft versus host diseaseGlecaprevir pibrentasvir 2 AbbVie hepatitis CCytarabine daunorubicin 2 Celator Pharmaceuticals acute myeloid leukemiaInotuzumab ozogamicin 2 Wyeth Pharmaceuticals B cell precursor acute lymphoblastic leukemiaDeutetrabenazine 2 Teva Pharmaceuticals tardive dyskinesiaAbemaciclib 2 Eli Lilly breast cancerAcalabrutinib 2 AstraZeneca UK mantle cell lymphomaVemurafenib 2 Hoffmann La Roche Erdheim Chester disease with BRAF V600 mutationAlectinib 2 Hoffmann La Roche ALK positive metastatic non small cell lung cancerLetermovir 2 Merck Sharp amp Dohme cytomegalovirus infectionBrentuximab vedotin 2 Seattle Genetics anaplastic large cell lymphomaEmicizumab kxwh 2 Genentech hemophiliaNivolumab 2 Bristol Myers Squibb melanoma2016 editDrug Manufacturer IndicationElbasvir grazoprevir 2 Merck Sharp amp Dohme Hepatitis CPalbociclib 2 Pfizer HR positive HER2 negative breast cancerCrizotinib 2 PF Prism ROS1 positive NSCLCVenetoclax 2 AbbVie CLL with 17p deletionCabozantinib 2 Exelixis renal cell carcinomaPimavanserin 2 Acadia Pharmaceuticals Parkinson s disease related psychosisIbrutinib 2 Pharmacyclics small lymphocytic lymphoma SLL with 17p deletionLenvatinib 2 Eisai renal cell carcinomaNivolumab 2 Bristol Myers Squibb Hodgkin lymphomaAtezolizumab 2 Genentech urothelial carcinomaSofosbuvir velpatasvir 2 Gilead Sciences chronic hepatitis C virus genotypes 1 2 3 4 5 or 6 infectionCanakinumab 2 Novartis Familial mediterranean feverCanakinumab 2 Novartis Hyper IgD syndromeCanakinumab 2 Novartis TNF receptor associated periodic syndromeLumacaftor ivacaftor 2 Vertex Pharmaceuticals cystic fibrosis with CFTR F508delAtezolizumab 2 Genentech metastatic NSCLCOlaratumab 2 Eli Lilly soft tissue sarcomaNivolumab 2 Bristol Myers Squibb head and neck cancerDaratumumab 2 Janssen multiple myelomaRucaparib 2 Clovis Oncology BRCA mutated ovarian cancer2015 editDrug Manufacturer IndicationIbrutinib 2 Pharmacyclics Waldenstrom s macroglobulinemiaPalbociclib 2 Pfizer ER positive HER2 negative breast cancerRanibizumab 2 Genentech diabetic retinopathyIvacaftor 2 Vertex Pharmaceuticals cystic fibrosis with a variety of CFTR mutationsAflibercept 2 Regeneron Pharmaceuticals diabetic retinopathySirolimus 2 PF Prism lymphangioleiomyomatosisSirolimus 2 PF Prism lymphangioleiomyomatosisLumacaftor ivacaftor 2 Vertex Pharmaceuticals cystic fibrosis with CFTR F508del mutationOmbitasvir paritaprevir ritonavir 2 AbbVie Hepatitis CUridine triacetate 2 Wellstat Therapeutics hereditary orotic aciduriaPembrolizumab 2 Merck Sharp amp Dohme PDL 1 positive NSCLCNivolumab 2 Bristol Myers Squibb metastatic NSCLCIdarucizumab 2 Boehringer Ingelheim reversal of dabigatranAsfotase alfa 2 Alexion Pharmaceuticals perinatal infantile childhood hypophosphatasiaOsimertinib 2 AstraZeneca EGFR positive NSCLCDaratumumab 2 Janssen Biotech multiple myelomaNivolumab 2 Bristol Myers Squibb advanced renal cell carcinomaElotuzumab 2 Bristol Myers Squibb multiple myelomaSebelipase alfa 2 Alexion Pharmaceuticals lysosomal acid lipase deficiencyAlectinib 2 Hoffmann La Roche ALK mutated NSCLCPembrolizumab 2 Merck Sharp amp Dohme metastatic melanoma2014 editDrug Manufacturer IndicationIvacaftor 2 Vertex Pharmaceuticals cystic fibrosis with CFTR mutationOfatumumab 2 Novartis Pharmaceuticals chronic lymphocytic leukemiaCeritinib 2 Novartis Pharmaceuticals ALK positive metastatic non small cell lung cancerIdelalisib 2 Gilead Sciences relapsed chronic lymphocytic leukemiaIbrutinib 2 Pharmacyclics chronic lymphocytic leukemia with 17p deletionEltrombopag 2 Novartis Pharmaceuticals aplastic anemiaPembrolizumab 2 Merck metastatic melanomaLedipasvir sofosbuvir 2 Gilead Sciences Hepatitis CPirfenidone 2 Genentech idiopathic pulmonary fibrosisNintedanib 2 Boehringer Ingelheim idiopathic pulmonary fibrosisBlinatumomab 2 Amgen acute lymphoblastic leukemiaOmbitasvir paritaprevir ritonavir dasabuvir 2 AbbVie Hepatitis CNivolumab 2 Bristol Myers Squibb 2 metastatic melanomaIvacaftor 2 Vertex Pharmaceuticals cystic fibrosis with CFTR R117H mutation2013 editDrug Manufacturer IndicationObinutuzumab 2 Genentech chronic lymphocytic leukemiaIbrutinib 2 Pharmacyclics mantle cell lymphomaSofosbuvir 2 Gilead Sciences Hepatitis CReferences edit a b c d e f g h i New Drug Therapy Approvals 2023 PDF U S Food and Drug Administration FDA Report January 2024 Archived from the original on 10 January 2024 Retrieved 9 January 2024 nbsp This article incorporates text from this source which is in the public domain a b c d e f g h i j k l m n o p q r s t u v w x y z aa ab ac ad ae af ag ah ai aj ak al am an ao ap aq ar as at au av aw ax ay az ba bb bc bd be bf bg bh bi bj bk bl bm bn bo bp bq br bs bt bu bv bw bx by bz ca cb cc cd ce cf cg ch ci cj ck cl cm cn co cp cq cr cs ct cu cv cw cx cy cz da db dc dd de df dg dh di dj dk dl dm dn do dp dq dr ds dt du dv dw dx dy dz ea eb ec ed ee ef eg eh ei ej ek el em en eo ep eq er es et eu ev ew ex ey ez fa fb fc fd fe ff fg fh fi CDER Breakthrough Therapy Designation Approvals Data as of December 31 2022 PDF U S Food and Drug Administration FDA Archived from the original on December 8 2019 Retrieved June 25 2023 nbsp This article incorporates text from this source which is in the public domain a b Breakthrough Therapy Approvals U S Food and Drug Administration FDA 2020 10 08 Archived from the original on 2023 06 11 Retrieved 2023 06 25 Approval report PDF U S Food and Drug Administration FDA Archived PDF from the original on 2019 04 24 Retrieved 2019 06 12 Retrieved from https en wikipedia org w index php title List of drugs granted breakthrough therapy designation amp oldid 1194685266, wikipedia, wiki, book, books, library,
