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.


2023

Brand name Manufacturer Indication
Elrexfio 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

Drug Manufacturer Indication
Abrocitinib Pfizer atopic dermatitis
Tebentafusp Immunocore HLA-A*02:01–positive uveal melanoma
Sutimlimab Bioverativ cold agglutinin disease
Lutetium (177Lu) vipivotide tetraxetan Novartis prostate-specific membrane antigen (PSMA)-positive metastatic castration-resistant

prostate cancer (mCRPC)

Alpelisib Novartis PIK3CA-related overgrowth spectrum conditions
Mavacamten Myokardia Inc class II-III obstructive hypertrophic cardiomyopathy
Trastuzumab 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 mutation
Dupilumab Regeneron Pharmaceuticals granted for two indications: eosinophilic esophagitis; and atopic dermatitis
Ivosidenib Servier Pharmaceuticals acute myeloid leukemia with a susceptible IDH1 mutation
Baricitinib Eli Lilly and Co severe alopecia areata
Setmelanotide Rhythm Pharmaceuticals Bardet–Biedl syndrome
Dextromethorphan/bupropion Axsome Therapeutics major depressive disorder
Pemigatinib Incyte myeloid/lymphoid neoplasms with FGFR1 rearrangement
Olipudase alfa Genzyme non–central nervous system manifestations of acid sphingomyelinase deficiency
Spesolimab Boehringer Ingelheim generalized pustular psoriasis
Futibatinib Taiho Oncology intrahepatic cholangiocarcinoma with FGFR2 gene fusions or other rearrangements
Teclistamab Janssen multiple myeloma
Teplizumab Provention Bio delay the onset of Stage 3 type 1 diabetes (T1D) in Stage 2 T1D
Atezolizumab Genentech alveolar soft part sarcoma
Adagrasib Mirati Therapeutics KRAS G12C-mutated non-small cell lung cancer
Mosunetuzumab Genentech follicular lymphoma
Lenacapavir Gilead Sciences HIV-1

2021

Drug Manufacturer Indication
Crizotinib PF Prism ALK-positive systemic anaplastic large cell lymphoma
Trastuzumab deruxtecan Daiichi Sankyo HER2-positive gastric or gastroesophageal junction adenocarcinoma, previously treated with trastuzumab
Umbralisib TG Therapeutics marginal zone lymphoma, previously treated with an anti-CD20-based regimen
Evinacumab Regeneron Pharmaceuticals homozygous familial hypercholesterolemia
Trilaciclib G1 Therapeutics myelosuppression, when administered prior to a platinum/etoposide or topotecan containing regimen for small cell lung cancer
Fosdenopterin Origin Biosciences molybdenum cofactor deficiency, Type A
Tocilizumab Genentech systemic sclerosis-associated interstitial lung disease
Rilonacept Kiniksa Pharmaceuticals recurrent pericarditis
Dostarlimab GSK mismatch repair deficient recurrent or advanced endometrial cancer
Dapagliflozin AstraZeneca chronic kidney disease
Bupivacaine/meloxicam Heron Therapeutics postsurgical analgesia
Amivantamab Janssen Biotech non-small cell lung cancer with EGFR exon 20 insertion mutations
Sotorasib Amgen non-small cell lung cancer with KRAS G12C mutation
Avapritinib Blueprint Medicines Corp granted for two indications: mast cell leukemia and advanced systemic mastocytosis
Belumosudil Kadmon Pharmaceuticals chronic graft-versus-host disease
Pembrolizumab Merck high risk, early-stage triple-negative breast cancer
Avalglucosidase alfa Genzyme late-onset Pompe disease
Lenvatinib Eisai Inc advanced renal cell carcinoma, used in combination with pembrolizumab
Difelikefalin Cara Therapeutics pruritis related to hemodialysis in chronic kidney disease
Mobocertinib Takeda Pharmaceuticals non-small cell lung cancer with EGFR exon 20 insertion mutations
Cabozantinib Exelixis thyroid cancer
Maralixibat Mirum Pharmaceuticals cholestatic pruritus in Alagille syndrome
Asciminib 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 mutation
Sirolimus AADI Bioscience metastatic or unresectable perivascular epithelioid cell tumor
Maribavir Takeda refractory post-transplant cytomegalovirus infection
Abatacept 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 donor
Voxelotor Global Blood Therapeutics Inc sickle cell disease
Cabotegravir ViiV Healthcare Co pre-exposure prophylaxis for HIV

2020

DrugManufacturerIndication
AvapritinibBlueprint Medicines Corpmetastatic gastrointestinal stromal tumor
Teprotumumab-trbwHorizon Therapeuticsthyroid eye disease
NintedanibBoehringer Ingelheimchronic fibrosing interstitial lung diseases
IpilimumabBristol-Myers Squibbhepatocellular carcinoma
NivolumabBristol-Myers Squibbhepatocellular carcinoma
SelumetinibAstraZenecaneurofibromatosis type 1
MitomycinUrogen Pharmaupper tract urothelial cancer
TucatinibSeattle Geneticsadvanced unresectable or metastatic HER2-positive breast cancer
PemigatinibIncyte Corpmetastatic cholangiocarcinoma with FGFR2 fusion
Sacituzumab govitecan-hziyImmunomedicsmetastatic triple-negative breast cancer
CapmatinibNovartismetastatic non-small cell lung cancer
SelpercatinibLoxo Oncologymetastatic RET fusion-positive non-small cell lung cancer
PomalidomideCelgene CorpAIDS-related Kaposi sarcoma
RucaparibClovis Oncologymetastatic castration-resistant prostate cancer
RipretinibDeciphera Pharmaceuticalsadvanced gastrointestinal stromal tumor
ArtesunateAmivas LLCsevere malaria
BevacizumabGenentechmetastatic hepatocellular carcinoma
AtezolizumabGenentechmetastatic hepatocellular carcinoma
Inebilizumab-cdonViela Bioneuromyelitis optica spectrum disorder
FostemsavirViiv Healthcaremultidrug-resistant HIV-1
Tafasitamab-cxixMorphoSysrelapsed or refractory diffuse large B-cell lymphoma
EsketamineJanssenmajor depressive disorder with acute suicidal ideation or behavior[2]
belantamab mafodotin-blmfGlaxoSmithKlinerefractory multiple myeloma
Satralizumab-mwgeGenentechneuromyelitis optica spectrum disorder
PralsetinibBlueprint Medicines CorpRET fusion-positive non-small cell lung cancer
PitolisantHarmony Biosciencescataplexy in adults with narcolepsy[2]
Atoltivimab/maftivimab/odesivimab-ebgnRegeneronEbola Zaire
LonafarnibEiger BioPharmaceuticalsHutchinson-Gilford progeria syndrome (HGPS), processing-deficient progeroid laminopathies with LMNA or ZMPSTE24 mutations[2]
LumasiranAlnylam Pharmaceuticalsprimary hyperoxaluria type 1[2]
Naxitamab-gqgkY-mAbs Therapeuticsrelapsed or refractory high-risk neuroblastoma, used in combination with GM-CSF
SetmelanotideRhythm Pharmaceuticalsmedical obesity caused by proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency
PralsetinibBlueprint Medicines CorpRET-mutant medullary thyroid cancer or advanced or metastatic RET fusion-positive thyroid cancer, in radioactive iodine-refractory patients[2]
BelimumabGlaxoSmithKlineactive lupus nephritis[2]
OsimertinibAstraZenecaadjuvant therapy after tumor resection in non-small cell lung cancer with EGFR exon 19 deletions or exon 21 L858R mutations
Ansuvimab-zyklRidgeback BiotherapeuticsEbola Zaire

2019

DrugManufacturerIndication
Letrozole/ribociclib[2]Novartis PharmaceuticalsHR-positive, HER2-negative advanced or metastatic breast cancer[2]
EsketamineJanssentreatment-resistant depression[2]
DupilumabRegeneron Pharmaceuticalsatopic dermatitis[2]
BrexanoloneSage Therapeuticspostpartum depression[2]
PalbociclibPfizermetastatic breast cancer[2]
ErdafitinibJanssenurothelial carcinoma[2]
PembrolizumabMerck Sharp & Dohmeadvanced renal cell carcinoma[2]
Ado-trastuzumab emtansineGenentechHER2-positive early breast cancer[2]
Tafamidis meglumineFoldrx Pharmaceuticalscardiomyopathy[2]
AvelumabEMD Seronoadvanced renal cell carcinoma[2]
VenetoclaxAbbViechronic lymphocytic leukemiaor small lymphocytic lymphoma[2]
GalcanezumabEli Lillyepisodic cluster headache[2]
Polatuzumab vedotin-piiqGenentechdiffuse large B-cell lymphoma[2]
PexidartinibDaiichi Sankyotenosynovial giant cell tumor[2]
EntrectinibGenentechsolid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion[2]
PembrolizumabMerckendometrial carcinoma[2]
LenvatinibEisaiendometrial carcinoma[2]
Elexacaftor/tezacaftor/ivacaftorVertex Pharmaceuticalscystic fibrosis[2]
ZanubrutinibBeiGenemantle cell lymphoma[2]
Crizanlizumab-tmcaNovartissickle cell disease[2]
GivosiranAlnylam Pharmaceuticalsacute hepatic porphyria[2]
AcalabrutinibAstraZenecarelapsed or refractory chronic lymphocytic leukemia[2]
AcalabrutinibAstraZenecauntreated chronic lymphocytic leukemia[2]
VoxelotorGlobal Blood Therapeuticssickle cell disease[2]
Enfortumab vedotin-ejfvAstellas Pharmametastatic urothelial carcinoma[2]
Fam-trastuzumab deruxtecan-nxkiDaiichi SankyoHER2-positive breast cancer[2]

[3]

2018

DrugManufacturerIndication
Tezacaftor/ivacaftor[2] Vertex Pharmaceuticals cystic fibrosis
Durvalumab[2] AstraZeneca UK unresectable Stage III non-small cell lung cancer
Ibalizumab-uiyk[2] Theratechnologies human immunodeficiency virus type 1 (HIV-1) infection
Brentuximab vedotin[2] Seattle Genetics Hodgkin lymphoma
Ipilimumab[2] Bristol-Myers Squibb renal cell carcinoma
Nivolumab[2] Bristol-Myers Squibb renal cell carcinoma
Burosumab-twza[2] Kyowa Hakko Kirin X-linked hypophosphatemia
Osimertinib[2] AstraZeneca metastatic non-small cell lung cancer
Dabrafenib[2] Novartis Pharmaceuticals melanoma with BRAF V600E or V600K mutations
Trametinib[2] Novartis Pharmaceuticals melanoma with BRAF V600E or V600K mutations
Dabrafenib[2] Novartis Pharmaceuticals anaplastic thyroid cancer with BRAF V600E mutation
Trametinib[2] Novartis Pharmaceuticals anaplastic thyroid cancer with BRAF V600E mutation
Fingolimod[2] Novartis Pharmaceuticals multiple sclerosis
Rituximab[2] Genentech pemphigus vulgaris
Venetoclax[2] AbbVie chronic lymphocytic leukemia
Pembrolizumab[2] Merck Sharp & Dohme mediastinal large B-cell lymphoma
Ipilimumab[2] Bristol-Myers Squibb metastatic colorectal cancer
Nivolumab[2] Bristol-Myers Squibb metastatic colorectal cancer
Ribociclib[2] Novartis Pharmaceuticals breast cancer
Tafenoquine[2] GlaxoSmithKline Plasmodium vivax malaria
Iobenguane I 131[2] Progenics Pharmaceuticals pheochromocytoma or paraganglioma
Lumacaftor/ivacaftor[2] Vertex Pharmaceuticals cystic fibrosis
Mogamulizumab-kpkc[2] Kyowa Hakko Kirin mycosis fungoides or Sézary syndrome
Patisiran[2] Alnylam Pharmaceuticals hereditary transthyretin-mediated amyloidosis
Cenegermin-bkbj[2] Dompe Farmaceutici neurotrophic keratitis
Lanadelumab-flyo[2] Takeda Pharmaceuticals hereditary angioedema
Cemiplimab-rwlc[2] Regeneron Pharmaceuticals cutaneous squamous cell carcinoma
Amikacin liposome inhalation suspension[2] Insmed mycobacterial lung disease
Emicizumab-kxwh[2] Genentech hemophilia
Lorlatinib[2] Pfizer ALK-positive, metastatic non-small cell lung cancer
Brentuximab vedotin[2] Seattle Genetics anaplastic large-cell lymphoma
Eltrombopag[2] Novartis Pharmaceuticals severe aplastic anemia
Emapalumab-lzsg[2] Swedish Orphan Biovitrum hemophagocytic lymphohistiocytosis
Venetoclax[2] AbbVie acute myeloid leukemia
Larotrectinib[2] Bayer Healthcare solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion
Larotrectinib[2] Bayer Healthcare solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion
Amifampridine[2] Catalyst Pharmaceuticals Lambert-Eaton myasthenic syndrome
Pembrolizumab[2] Merck Sharp & Dohme Merkel cell carcinoma
Tagraxofusp-erzs[2] Stemline Therapeutics blastic plasmacytoid dendritic cell neoplasm

[3]

2017

[4]

DrugManufacturerIndication
Nivolumab[2]Bristol-Myers Squibburothelial carcinoma
Ribociclib[2]Novartis PharmaceuticalsHR-positive, HER2-negative breast cancer
Pembrolizumab[2]Merck Sharp & DohmeHodgkin Lymphoma
Avelumab[2]EMD Seronometastatic Merkel cell carcinoma
Niraparib[2]GlaxoSmithKlinerecurrent ovarian cancer responsive to platinum-based chemotherapy
Ocrelizumab[2]Genentechmultiple sclerosis
Dupilumab[2]Regeneron Pharmaceuticalsatopic dermatitis
Palbociclib[2]PfizerHR-positive, HER2-negative breast cancer
Valbenazine[2]Neurocrine Biosciencestardive dyskinesia
Ranibizumab[2]Genentechdiabetic retinopathy
Cerliponase alfa[2]BioMarin Pharmaceuticaltripeptidyl peptidase 1 (TPP1) deficiency
Midostaurin[2]Novartis PharmaceuticalsFLT3-positive acute myeloid leukemia
Brigatinib[2]Takeda PharmaceuticalsALK-positive non-small cell lung cancer
Durvalumab[2]AstraZeneca UKmetastatic urothelial carcinoma
Letrozole/ribociclib[2]Novartis Pharmaceuticalsbreast cancer
Pembrolizumab[2]Merck Sharp & Dohmeurothelial carcinoma
Tocilizumab[2]Genentechgiant cell arteritis
Pembrolizumab[2]Merck Sharp & Dohmemicrosatellite instability-high tumors
Ceritinib[2]Novartis PharmaceuticalsALK-positive, metastatic non-small cell lung cancer
Dabrafenib[2]Novartis Pharmaceuticalsmetastatic non-small cell lung cancer with BRAF V600E mutation
Trametinib[2]Novartis Pharmaceuticalsmetastatic non-small cell lung cancer with BRAF V600E mutation
Ibrutinib[2]Pharmacyclicschronic graft-versus-host disease
Glecaprevir/pibrentasvir[2]AbbViehepatitis C
Cytarabine/daunorubicin[2]Celator Pharmaceuticalsacute myeloid leukemia
Inotuzumab ozogamicin[2]Wyeth PharmaceuticalsB-cell precursor acute lymphoblastic leukemia
Deutetrabenazine[2]Teva Pharmaceuticalstardive dyskinesia
Abemaciclib[2]Eli Lillybreast cancer
Acalabrutinib[2]AstraZeneca UKmantle cell lymphoma
Vemurafenib[2]Hoffmann-La RocheErdheim-Chester disease with BRAF V600 mutation
Alectinib[2]Hoffmann-La RocheALK-positive, metastatic non-small cell lung cancer
Letermovir[2]Merck Sharp & Dohmecytomegalovirus infection
Brentuximab vedotin[2]Seattle Geneticsanaplastic large-cell lymphoma
Emicizumab-kxwh[2]Genentechhemophilia
Nivolumab[2]Bristol-Myers Squibbmelanoma

2016

DrugManufacturerIndication
Elbasvir/grazoprevir[2]Merck Sharp & DohmeHepatitis C
Palbociclib[2]PfizerHR-positive, HER2-negative breast cancer
Crizotinib[2]PF PrismROS1-positive NSCLC
Venetoclax[2]AbbVieCLL with 17p deletion
Cabozantinib[2]Exelixisrenal cell carcinoma
Pimavanserin[2]Acadia PharmaceuticalsParkinson's disease-related psychosis
Ibrutinib[2]Pharmacyclicssmall lymphocytic lymphoma (SLL) with 17p deletion
Lenvatinib[2]Eisairenal cell carcinoma
Nivolumab[2]Bristol-Myers SquibbHodgkin lymphoma
Atezolizumab[2]Genentechurothelial carcinoma
Sofosbuvir/velpatasvir[2]Gilead Scienceschronic hepatitis C virus genotypes 1, 2, 3, 4, 5 or 6 infection
Canakinumab[2]NovartisFamilial mediterranean fever
Canakinumab[2]NovartisHyper-IgD syndrome
Canakinumab[2]NovartisTNF receptor associated periodic syndrome
Lumacaftor/ivacaftor[2]Vertex Pharmaceuticalscystic fibrosis with CFTR F508del
Atezolizumab[2]Genentechmetastatic NSCLC
Olaratumab[2]Eli Lillysoft-tissue sarcoma
Nivolumab[2]Bristol-Myers Squibbhead and neck cancer
Daratumumab[2]Janssenmultiple myeloma
Rucaparib[2]Clovis OncologyBRCA-mutated ovarian cancer

2015

DrugManufacturerIndication
Ibrutinib[2]PharmacyclicsWaldenstrom’s macroglobulinemia
Palbociclib[2]PfizerER-positive, HER2-negative breast cancer
Ranibizumab[2]Genentechdiabetic retinopathy
Ivacaftor[2]Vertex Pharmaceuticalscystic fibrosis with a variety of CFTR mutations
Aflibercept[2]Regeneron Pharmaceuticalsdiabetic retinopathy
Sirolimus[2]PF Prismlymphangioleiomyomatosis
Sirolimus[2]PF Prismlymphangioleiomyomatosis
Lumacaftor/ivacaftor[2]Vertex Pharmaceuticalscystic fibrosis with CFTR F508del mutation
Ombitasvir/paritaprevir/ritonavir[2]AbbVieHepatitis C
Uridine triacetate[2]Wellstat Therapeuticshereditary orotic aciduria
Pembrolizumab[2]Merck Sharp & DohmePDL-1 positive NSCLC
Nivolumab[2]Bristol-Myers Squibbmetastatic NSCLC
Idarucizumab[2]Boehringer Ingelheimreversal of dabigatran
Asfotase alfa[2]Alexion Pharmaceuticalsperinatal/infantile/childhood hypophosphatasia
Osimertinib[2]AstraZenecaEGFR-positive NSCLC
Daratumumab[2]Janssen Biotechmultiple myeloma
Nivolumab[2]Bristol-Myers Squibbadvanced renal cell carcinoma
Elotuzumab[2]Bristol-Myers Squibbmultiple myeloma
Sebelipase alfa[2]Alexion Pharmaceuticalslysosomal acid lipase deficiency
Alectinib[2]Hoffmann-La RocheALK-mutated NSCLC
Pembrolizumab[2]Merck Sharp & Dohmemetastatic melanoma

2014

DrugManufacturerIndication
Ivacaftor[2]Vertex Pharmaceuticalscystic fibrosis with CFTR mutation
Ofatumumab[2]Novartis Pharmaceuticalschronic lymphocytic leukemia
Ceritinib[2]Novartis PharmaceuticalsALK-positive metastatic non-small cell lung cancer
Idelalisib[2]Gilead Sciencesrelapsed chronic lymphocytic leukemia
Ibrutinib[2]Pharmacyclicschronic lymphocytic leukemia (with 17p deletion
Eltrombopag[2]Novartis Pharmaceuticalsaplastic anemia
Pembrolizumab[2]Merckmetastatic melanoma
Ledipasvir/sofosbuvir[2]Gilead SciencesHepatitis C
Pirfenidone[2]Genentechidiopathic pulmonary fibrosis
Nintedanib[2]Boehringer Ingelheimidiopathic pulmonary fibrosis
Blinatumomab[2]Amgenacute lymphoblastic leukemia
Ombitasvir/paritaprevir/ritonavir/dasabuvir[2]AbbVieHepatitis C
Nivolumab[2]Bristol-Myers Squibb[2]metastatic melanoma
Ivacaftor[2]Vertex Pharmaceuticalscystic fibrosis with CFTR R117H mutation

2013

DrugManufacturerIndication
Obinutuzumab[2]Genentechchronic lymphocytic leukemia
Ibrutinib[2]Pharmacyclicsmantle cell lymphoma
Sofosbuvir[2]Gilead SciencesHepatitis C

References

  1. 1 2 3 4 5 6 7 8 9 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. Public Domain This article incorporates text from this source, which is in the public domain.
  2. 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 20 21 22 23 24 25 26 27 28 29 30 31 32 33 34 35 36 37 38 39 40 41 42 43 44 45 46 47 48 49 50 51 52 53 54 55 56 57 58 59 60 61 62 63 64 65 66 67 68 69 70 71 72 73 74 75 76 77 78 79 80 81 82 83 84 85 86 87 88 89 90 91 92 93 94 95 96 97 98 99 100 101 102 103 104 105 106 107 108 109 110 111 112 113 114 115 116 117 118 119 120 121 122 123 124 125 126 127 128 129 130 131 132 133 134 135 136 137 138 139 140 141 142 143 144 145 146 147 148 149 150 151 152 153 154 155 156 157 158 159 160 161 162 163 164 165 "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. Public Domain This article incorporates text from this source, which is in the public domain.
  3. 1 2 "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.
  4. ↑ "Approval report" (PDF). U.S. Food and Drug Administration (FDA). Archived (PDF) from the original on 2019-04-24. Retrieved 2019-06-12.
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