List of drugs granted breakthrough therapy designation
Appearance
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.[1]
2024
[edit]Brand name | Manufacturer | Indication |
---|---|---|
Anktiva | Indicated with Bacillus Calmette-Guérin (BCG) for the treatment of adults with BCG-unresponsive nonmuscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors[2] | |
Augtyro | Treatment of people aged 12 years of age and older with solid tumors that: have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion, are locally advanced or metastatic or where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory alternative therapy[2] | |
Beqvez | Pfizer | Treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who are receiving routine prophylaxis and without pre-existing neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid detected by an FDA-approved test.[3] |
Blincyto | Treatment of CD19-positive Philadelphia chromosome-negative B- cell precursor acute lymphoblastic leukemia (ALL) in the consolidation phase of multiphase chemotherapy in people aged one month of age and older[2] | |
Carvykti | Janssen Biotech | Treatment of adults with relapsed or refractory multiple myeloma, who previously received a proteasome inhibitor (PI), an immunomodulatory agent (IMiD) and an anti-CD38 antibody[3] |
Dupixent | As an add-on maintenance treatment in adults with inadequately controlled chronic obstructive pulmonary disease (COPD) and an eosinophilic phenotype[2] | |
Enhertu | Treatment of adults with unresectable or metastatic HER2- positive (IHC 3+) solid tumors who have received prior systemic treatment and have no satisfactory alternative treatment options[2] | |
Eohilia | For 12 weeks of treatment in people aged 11 years of age and older with eosinophilic esophagitis[2] | |
Epkinly | Treatment of adults with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy[2] | |
Imdelltra | Treatment of adults with extensive stage small cell lung cancer (ES-SCLC) with disease progression on or after platinum-based chemotherapy[2] | |
Iqirvo | Treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in people unable to tolerate UDCA[2] | |
Kisunla | Treatment of Alzheimer’s disease[2] | |
Krazati | In combination with cetuximab for the treatment of adults with KRAS G12C mutated locally advanced or metastatic colorectal cancer who have been previously treated with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy[2] | |
Livdelzi | Treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in people unable to tolerate UDCA[2] | |
Miplyffa | In combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in people aged 2 years of age and older[2] | |
Nemluvio | Treatment of adults with Prurigo Nodularis[2] | |
Ojemda oral suspension | Treatment of people aged 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation[2] | |
Ojemda tablet | Treatment of people aged 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation[2] | |
Rezdiffra | In conjunction with diet and exercise, for the treatment of adults with noncirrhotic nonalcoholic steatohepatitis (NASH) with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis)[2] | |
Spevigo | Treatment of generalized pustular psoriasis (GPP) in people aged 12 years of age and older and weighing at least 40 kg[2] | |
Tagrisso | In combination with pemetrexed and platinum-based chemotherapy for the first-line treatment of adults with locally advanced or metastatic non- small cell lung cancer whose tumors have EGFR exon 19 deletions or exon 21 L858R mutations, as detected by an FDA-approved test[2] | |
Tagrisso | Treatment of adults with locally advanced, unresectable (stage III) non- small cell lung cancer (NSCLC) whose disease has not progressed during or following concurrent or sequential platinum-based chemoradiation therapy and whose tumors have EGFR exon 19 deletions or exon 21 L858R mutations, as detected by an FDA-approved test[2] | |
Voranigo | Treatment of people aged 12 years of age and older with Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation, following surgery including biopsy, sub- total resection, or gross total resection[2] | |
Voydeya | As add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH)[2] | |
Winrevair | Treatment of adults with pulmonary arterial hypertension (PAH, World Health Organization [WHO] Group 1) to increase exercise capacity, improve WHO functional class (FC), and reduce the risk of clinical worsening events[2] | |
Xolair | Treatment of IgE-mediated food allergy in people aged 1 year of age and older for the reduction of allergic reactions (Type I), including anaphylaxis, that may occur with accidental exposure to one or more foods. To be used in conjunction with food allergen avoidance[2] |
2023
[edit]Brand name | Manufacturer | Indication |
---|---|---|
Elrexfio | To treat relapsed or refractory multiple myeloma after at least four lines of therapy[4] | |
Fabhalta | To treat paroxysmal nocturnal hemoglobinuria[4] | |
Izervay | To treat geographic atrophy secondary to age-related macular degeneration[4] | |
Leqembi | To treat Alzheimer's disease[4] | |
Loqtorzi | To treat recurrent or metastatic nasopharyngeal carcinoma with or following other therapies[4] | |
Ogsiveo | To treat desmoid tumors[4] | |
Pombiliti | To treat late-onset Pompe disease with miglustat[4] | |
Rivfloza | To lower urinary oxalate levels in primary hyperoxaluria type 1 and relatively preserved kidney function[4] | |
Talvey | To treat relapsed or refractory multiple myeloma after at least four therapies[4] |
2022
[edit]2021
[edit]2020
[edit]2019
[edit]2018
[edit]2017
[edit]2016
[edit]2015
[edit]2014
[edit]2013
[edit]Drug | Manufacturer | Indication |
---|---|---|
Obinutuzumab[1] | Genentech | chronic lymphocytic leukemia |
Ibrutinib[1] | Pharmacyclics | mantle cell lymphoma |
Sofosbuvir[1] | Gilead Sciences | Hepatitis C |
References
[edit]- ^ 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 fj "CDER Breakthrough Therapy Designation Approvals" (PDF). U.S. Food and Drug Administration (FDA). December 31, 2023. Archived from the original on December 7, 2024. Retrieved June 25, 2023. 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 "CY 2024 CDER Breakthrough Therapy Calendar Year Approvals" (PDF). U.S. Food and Drug Administration (FDA). September 30, 2024. This article incorporates text from this source, which is in the public domain.
- ^ a b "CY2024 CBER Breakthrough Therapy Approvals" (PDF). U.S. Food and Drug Administration (FDA). June 30, 2024. This article incorporates text from this source, which is in the public domain.
- ^ 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 January 10, 2024. Retrieved January 9, 2024. 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). October 8, 2020. Archived from the original on June 11, 2023. Retrieved June 25, 2023.
- ^ "FDA grants Breakthrough Therapy Designation for Roche's balovaptan in autism spectrum disorder". Roche. Retrieved April 8, 2024.
- ^ "Approval report" (PDF). U.S. Food and Drug Administration (FDA). Archived (PDF) from the original on April 24, 2019. Retrieved June 12, 2019.