Wikipedia

List of drugs granted breakthrough therapy designation

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

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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

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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

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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

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Drug Manufacturer Indication
Avapritinib Blueprint Medicines Corp metastatic gastrointestinal stromal tumor
Teprotumumab-trbw Horizon Therapeutics thyroid eye disease
Nintedanib Boehringer Ingelheim chronic fibrosing interstitial lung diseases
Ipilimumab Bristol-Myers Squibb hepatocellular carcinoma
Nivolumab Bristol-Myers Squibb hepatocellular carcinoma
Selumetinib AstraZeneca neurofibromatosis type 1
Mitomycin Urogen Pharma upper tract urothelial cancer
Tucatinib Seattle Genetics advanced unresectable or metastatic HER2-positive breast cancer
Pemigatinib Incyte Corp metastatic cholangiocarcinoma with FGFR2 fusion
Sacituzumab govitecan-hziy Immunomedics metastatic triple-negative breast cancer
Capmatinib Novartis metastatic non-small cell lung cancer
Selpercatinib Loxo Oncology metastatic RET fusion-positive non-small cell lung cancer
Pomalidomide Celgene Corp AIDS-related Kaposi sarcoma
Rucaparib Clovis Oncology metastatic castration-resistant prostate cancer
Ripretinib Deciphera Pharmaceuticals advanced gastrointestinal stromal tumor
Artesunate Amivas LLC severe malaria
Bevacizumab Genentech metastatic hepatocellular carcinoma
Atezolizumab Genentech metastatic hepatocellular carcinoma
Inebilizumab-cdon Viela Bio neuromyelitis optica spectrum disorder
Fostemsavir Viiv Healthcare multidrug-resistant HIV-1
Tafasitamab-cxix MorphoSys relapsed or refractory diffuse large B-cell lymphoma
Esketamine Janssen major depressive disorder with acute suicidal ideation or behavior[2]
belantamab mafodotin-blmf GlaxoSmithKline refractory multiple myeloma
Satralizumab-mwge Genentech neuromyelitis optica spectrum disorder
Pralsetinib Blueprint Medicines Corp RET fusion-positive non-small cell lung cancer
Pitolisant Harmony Biosciences cataplexy in adults with narcolepsy[2]
Atoltivimab/maftivimab/odesivimab-ebgn Regeneron Ebola Zaire
Lonafarnib 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-CSF
Setmelanotide Rhythm Pharmaceuticals medical obesity caused by proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency
Pralsetinib 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 mutations
Ansuvimab-zykl Ridgeback Biotherapeutics Ebola Zaire

2019

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Drug Manufacturer Indication
Letrozole/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 & 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

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Drug Manufacturer Indication
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
Balovaptan[4] Roche Autism spectrum

[3]

2017

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[5]

Drug Manufacturer Indication
Nivolumab[2] Bristol-Myers Squibb urothelial carcinoma
Ribociclib[2] Novartis Pharmaceuticals HR-positive, HER2-negative breast cancer
Pembrolizumab[2] Merck Sharp & Dohme Hodgkin Lymphoma
Avelumab[2] EMD Serono metastatic Merkel cell carcinoma
Niraparib[2] GlaxoSmithKline recurrent ovarian cancer responsive to platinum-based chemotherapy
Ocrelizumab[2] Genentech multiple sclerosis
Dupilumab[2] Regeneron Pharmaceuticals atopic dermatitis
Palbociclib[2] Pfizer HR-positive, HER2-negative breast cancer
Valbenazine[2] Neurocrine Biosciences tardive dyskinesia
Ranibizumab[2] Genentech diabetic retinopathy
Cerliponase alfa[2] BioMarin Pharmaceutical tripeptidyl peptidase 1 (TPP1) deficiency
Midostaurin[2] Novartis Pharmaceuticals FLT3-positive acute myeloid leukemia
Brigatinib[2] Takeda Pharmaceuticals ALK-positive non-small cell lung cancer
Durvalumab[2] AstraZeneca UK metastatic urothelial carcinoma
Letrozole/ribociclib[2] Novartis Pharmaceuticals breast cancer
Pembrolizumab[2] Merck Sharp & Dohme urothelial carcinoma
Tocilizumab[2] Genentech giant cell arteritis
Pembrolizumab[2] Merck Sharp & Dohme microsatellite instability-high tumors
Ceritinib[2] Novartis Pharmaceuticals ALK-positive, metastatic non-small cell lung cancer
Dabrafenib[2] Novartis Pharmaceuticals metastatic non-small cell lung cancer with BRAF V600E mutation
Trametinib[2] Novartis Pharmaceuticals metastatic non-small cell lung cancer with BRAF V600E mutation
Ibrutinib[2] Pharmacyclics chronic graft-versus-host disease
Glecaprevir/pibrentasvir[2] AbbVie hepatitis C
Cytarabine/daunorubicin[2] Celator Pharmaceuticals acute myeloid leukemia
Inotuzumab ozogamicin[2] Wyeth Pharmaceuticals B-cell precursor acute lymphoblastic leukemia
Deutetrabenazine[2] Teva Pharmaceuticals tardive dyskinesia
Abemaciclib[2] Eli Lilly breast cancer
Acalabrutinib[2] AstraZeneca UK mantle cell lymphoma
Vemurafenib[2] Hoffmann-La Roche Erdheim-Chester disease with BRAF V600 mutation
Alectinib[2] Hoffmann-La Roche ALK-positive, metastatic non-small cell lung cancer
Letermovir[2] Merck Sharp & Dohme cytomegalovirus infection
Brentuximab vedotin[2] Seattle Genetics anaplastic large-cell lymphoma
Emicizumab-kxwh[2] Genentech hemophilia
Nivolumab[2] Bristol-Myers Squibb melanoma

2016

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Drug Manufacturer Indication
Elbasvir/grazoprevir[2] Merck Sharp & Dohme Hepatitis C
Palbociclib[2] Pfizer HR-positive, HER2-negative breast cancer
Crizotinib[2] PF Prism ROS1-positive NSCLC
Venetoclax[2] AbbVie CLL with 17p deletion
Cabozantinib[2] Exelixis renal cell carcinoma
Pimavanserin[2] Acadia Pharmaceuticals Parkinson's disease-related psychosis
Ibrutinib[2] Pharmacyclics small lymphocytic lymphoma (SLL) with 17p deletion
Lenvatinib[2] Eisai renal cell carcinoma
Nivolumab[2] Bristol-Myers Squibb Hodgkin lymphoma
Atezolizumab[2] Genentech urothelial carcinoma
Sofosbuvir/velpatasvir[2] Gilead Sciences chronic hepatitis C virus genotypes 1, 2, 3, 4, 5 or 6 infection
Canakinumab[2] Novartis Familial mediterranean fever
Canakinumab[2] Novartis Hyper-IgD syndrome
Canakinumab[2] Novartis TNF receptor associated periodic syndrome
Lumacaftor/ivacaftor[2] Vertex Pharmaceuticals cystic fibrosis with CFTR F508del
Atezolizumab[2] Genentech metastatic NSCLC
Olaratumab[2] Eli Lilly soft-tissue sarcoma
Nivolumab[2] Bristol-Myers Squibb head and neck cancer
Daratumumab[2] Janssen multiple myeloma
Rucaparib[2] Clovis Oncology BRCA-mutated ovarian cancer

2015

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Drug Manufacturer Indication
Ibrutinib[2] Pharmacyclics Waldenstrom’s macroglobulinemia
Palbociclib[2] Pfizer ER-positive, HER2-negative breast cancer
Ranibizumab[2] Genentech diabetic retinopathy
Ivacaftor[2] Vertex Pharmaceuticals cystic fibrosis with a variety of CFTR mutations
Aflibercept[2] Regeneron Pharmaceuticals diabetic retinopathy
Sirolimus[2] PF Prism lymphangioleiomyomatosis
Sirolimus[2] PF Prism lymphangioleiomyomatosis
Lumacaftor/ivacaftor[2] Vertex Pharmaceuticals cystic fibrosis with CFTR F508del mutation
Ombitasvir/paritaprevir/ritonavir[2] AbbVie Hepatitis C
Uridine triacetate[2] Wellstat Therapeutics hereditary orotic aciduria
Pembrolizumab[2] Merck Sharp & Dohme PDL-1 positive NSCLC
Nivolumab[2] Bristol-Myers Squibb metastatic NSCLC
Idarucizumab[2] Boehringer Ingelheim reversal of dabigatran
Asfotase alfa[2] Alexion Pharmaceuticals perinatal/infantile/childhood hypophosphatasia
Osimertinib[2] AstraZeneca EGFR-positive NSCLC
Daratumumab[2] Janssen Biotech multiple myeloma
Nivolumab[2] Bristol-Myers Squibb advanced renal cell carcinoma
Elotuzumab[2] Bristol-Myers Squibb multiple myeloma
Sebelipase alfa[2] Alexion Pharmaceuticals lysosomal acid lipase deficiency
Alectinib[2] Hoffmann-La Roche ALK-mutated NSCLC
Pembrolizumab[2] Merck Sharp & Dohme metastatic melanoma

2014

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Drug Manufacturer Indication
Ivacaftor[2] Vertex Pharmaceuticals cystic fibrosis with CFTR mutation
Ofatumumab[2] Novartis Pharmaceuticals chronic lymphocytic leukemia
Ceritinib[2] Novartis Pharmaceuticals ALK-positive metastatic non-small cell lung cancer
Idelalisib[2] Gilead Sciences relapsed chronic lymphocytic leukemia
Ibrutinib[2] Pharmacyclics chronic lymphocytic leukemia (with 17p deletion
Eltrombopag[2] Novartis Pharmaceuticals aplastic anemia
Pembrolizumab[2] Merck metastatic melanoma
Ledipasvir/sofosbuvir[2] Gilead Sciences Hepatitis C
Pirfenidone[2] Genentech idiopathic pulmonary fibrosis
Nintedanib[2] Boehringer Ingelheim idiopathic pulmonary fibrosis
Blinatumomab[2] Amgen acute lymphoblastic leukemia
Ombitasvir/paritaprevir/ritonavir/dasabuvir[2] AbbVie Hepatitis C
Nivolumab[2] Bristol-Myers Squibb[2] metastatic melanoma
Ivacaftor[2] Vertex Pharmaceuticals cystic fibrosis with CFTR R117H mutation

2013

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Drug Manufacturer Indication
Obinutuzumab[2] Genentech chronic lymphocytic leukemia
Ibrutinib[2] Pharmacyclics mantle cell lymphoma
Sofosbuvir[2] Gilead Sciences Hepatitis C

References

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  1. ^ 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.   This article incorporates text from this source, which is in the public domain.
  2. ^ 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.   This article incorporates text from this source, which is in the public domain.
  3. ^ 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.
  4. ^ "FDA grants Breakthrough Therapy Designation for Roche's balovaptan in autism spectrum disorder". Roche. Retrieved 2024-04-08.
  5. ^ "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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