List of drugs granted breakthrough therapy designation
From Wikipedia, the free encyclopedia
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]
| Drug | Manufacturer | Indication |
|---|---|---|
| Aficamten | The treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms[2] | |
| Avutometinib | The treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy[2] | |
| Brensocatib | The treatment of non-cystic fibrosis bronchiectasis (NCFB) in adult and pediatric patients 12 years of age and older[2] | |
| Datopotamab deruxtecan | The treatment of adult patients with locally advanced or metastatic epidermal growth factor receptor (EGFR) mutated non -small cell lung cancer (NSCLC) who have received prior EGFR- directed therapy and platinum- based therapy[2] | |
| Diazoxide choline | The treatment of hyperphagia in adults and pediatric patients 4 years of age and older with Prader-Willi syndrome[2] | |
| Doxecitine/doxribtimine | Treatment of thymidine kinase 2 deficiency (TK2d) in adults and pediatric patients with an age of symptom onset on or before 12 years[2] | |
| Durvalumab | In combination with fluorouracil, leucovorin, oxaliplatin and docetaxel (FLOT) as neoadjuvant and adjuvant treatment, followed by single-agent IMFINZI, is indicated for the treatment of adult patients with resectable gastric or gastroesophageal junction adenocarcinoma (GC/GEJC)[2] | |
| Epcoritamab | In combination with lenalidomide and rituximab for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL)[2] | |
| Esketamine | For treatment-resistant depression (TRD) in adults, as monotherapy or in conjunction with an oral antidepressant[2] | |
| Evinacumab | As an adjunct to diet and exercise and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies to reduce LDL-C in adults and pediatric patients, aged 1 year and older, with homozygous familial hypercholesterolemia (HoFH)[2] | |
| Trastuzumab deruxtecan | The treatment of adult patients with unresectable or metastatic hormone receptor (HR)-positive, HER2-low (IHC 1+ or IHC 2+/ISH-) or HER2-ultralow (IHC 0 with membrane staining) breast cancer, as determined by an FDA-approved test, that has progressed on one or more endocrine therapies in the metastatic setting[2] | |
| Trastuzumab deruxtecan | In combination with pertuzumab for the first-line treatment of adult patients with unresectable or metastatic HER2-positive (IHC 3+ or ISH+) breast cancer as determined by an FDA-approved test[2] | |
| Gemcitabine-Releasing Intravesical System | Treatment of adult patients with Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (HR-NMIBC) with carcinoma in situ (CIS) with or without papillary tumors[2] | |
| Glecaprevir/pibrentasvir | The treatment of acute hepatitis C virus (HCV) infection in adults and pediatric patients ages 3 to less than 18 years of age[2] | |
| Inebilizumab | For use of in adult patients with immunoglobulin G4- Related Disease (IgG4-RD)[2] | |
| Ipilimumab | In combination with nivolumab, is indicated for the treatment of adult and pediatric patients 12 years and older with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer (CRC)[2] | |
| Iptacopan | The treatment of adults with complement 3 glomerulopathy (C3G), to reduce proteinuria[2] | |
| Lenacapavir | Pre-exposure prophylaxis (PrEP) to reduce the risk of sexually acquired HIV-1 in adults and adolescents weighing at least 35 kg who are at risk for HIV-1 acquisition[2] | |
| Narsoplimab | Treatment of adult and pediatric patients two years of age and older with hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA)[2] | |
| Nerandomilast | The treatment of idiopathic pulmonary fibrosis in adult patients[2] | |
| Nerandomilast | The treatment of progressive pulmonary fibrosis in adult patients[2] | |
| Nivolumab | In combination with ipilimumab, is indicated for the treatment of adult and pediatric patients 12 years and older with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer (CRC)[2] | |
| Obinutuzumab | Treatment of adult patients with active lupus nephritis (LN) who are receiving standard therapy A short duration infusion of approximately 90 minutes from the second infusion onwards in patients with active lupus nephritis (LN) who have completed the first obinutuzumab administration at the standard infusion rate without experiencing any Grade = 3 infusion-related reactions (IRRs)[2] | |
| Penpulimab | Penpulimab-kcqx is indicated: • In combination with either cisplatin or carboplatin and gemcitabine, for the firstline treatment of adults with recurrent or metastatic non-keratinizing nasopharyngeal carcinoma (NPC) • As a single agent, for the treatment of adults with metastatic non-keratinizing NPC with disease progression on or after platinum-based chemotherapy and at least one other prior line of therapy[2] | |
| Plozasiran | As an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS)[2] | |
| Semaglutide | For the treatment of noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH), formerly known as nonalcoholic steatohepatitis (NASH), with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis) in adults[2] | |
| Sevabertinib | Treatment of adult patients with locally advanced or metastatic non-squamous, non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) tyrosine kinase domain activating mutations, as detected by an FDA-approved test, and who have received a prior systemic therapy[2] | |
| Sibeprenlimab | To reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk for disease progression[2] | |
| Sotorasib | In combination with panitumumab, for the treatment of adult patients with KRAS G12C-mutated metastatic colorectal cancer as determined by an FDA approved test, who have received prior fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy[2] | |
| Sunvozertinib | The treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations, as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy[2] | |
| Suzetrigine | The treatment of moderate to severe acute pain in adults[2] | |
| Taletrectinib | The treatment of adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC)[2] | |
| Telisotuzumab vedotin | The treatment of adult patients with locally advanced or metastatic, non-squamous non-small cell lung cancer (NSCLC) with high c-Met protein overexpression [>=50% of tumor cells with strong (3+) staining], as determined by an FDA-approved test, who have received a prior systemic therapy[2] | |
| Ziftomenib | Treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation who have no satisfactory alternative treatment options[2] | |
| Zongertinib | Treatment of adult patients with unresectable or metastatic non-squamous non-small cell lung cancer (NSCLC) whose tumors have HER2 (ERBB2) tyrosine kinase domain activating mutations, as detected by an FDA-approved test, and who have received prior systemic therapy[2] |
2024
| Drug | Manufacturer | Indication |
|---|---|---|
| Adagrasib | 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[3] | |
| Arimoclomol | In combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in people aged two years of age and older[3] | |
| Asciminib | Novartis | Treatment of adults with newly diagnosed Philadelphia chromosomepositive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP)[3] |
| Blinatumomab | 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[3] | |
| Budesonide | For twelve weeks of treatment in people aged eleven years of age and older with eosinophilic esophagitis[3] | |
| Ciltacabtagene autoleucel | 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[4] |
| Crinecerfont (capsules) | Neurocrine Biosciences | Adjunctive treatment to glucocorticoid replacement to control androgens in people aged four years of age and older with classic congenital adrenal hyperplasia (CAH)[3] |
| Crinecerfont (solution) | Neurocrine Biosciences | Adjunctive treatment to glucocorticoid replacement to control androgens in people aged four years of age and older with classic congenital adrenal hyperplasia (CAH)[3] |
| Danicopan | As add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH)[3] | |
| Donanemab | Treatment of Alzheimer’s disease[3] | |
| Dupilumab | Regeneron | As an add-on maintenance treatment in adults with inadequately controlled chronic obstructive pulmonary disease (COPD) and an eosinophilic phenotype[3] |
| Durvalumab | Astrazeneca | Treatment of adults with limited stage small cell lung cancer (LS-SCLC) whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy (cCRT)[3] |
| Elafibranor | 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[3] | |
| Epkinly | Treatment of adults with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy[3] | |
| Fidanacogene elaparvovec | 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.[4] |
| Inavolisib | Genentech | In combination with palbociclib and fulvestrant for the treatment of adults with endocrine-resistant, PIK3CA-mutated, hormone receptor (HR)-positive, human epidermal growth-factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer, as detected by an FDA-approved test, following recurrence on or after completing adjuvant endocrine therapy[3] |
| Nemolizumab | Treatment of adults with Prurigo Nodularis[3] | |
| Nogapendekin alfa inbakicept | 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[3] | |
| Olezarsen | Ionis Pharmaceuticals | Adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS)[3] |
| Omalizumab | Treatment of IgE-mediated food allergy in people aged one 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[3] | |
| Osimertinib | 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[3] | |
| Osimertinib | 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[3] | |
| Repotrectinib | Treatment of people aged twelve 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[3] | |
| Resmetirom | 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)[3] | |
| Revumenib | Treatment of relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in people aged one year of age and older[3] | |
| Seladelpar | 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[3] | |
| Sotatercept | 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[3] | |
| Spesolimab | Treatment of generalized pustular psoriasis (GPP) in people aged twelve years of age and older and weighing at least 40 kg[3] | |
| Tarlatamab | Treatment of adults with extensive stage small cell lung cancer (ES-SCLC) with disease progression on or after platinum-based chemotherapy[3] | |
| Tirzepatide | Eli Lilly | Treatment of moderate to severe obstructive sleep apnea (OSA) in adults with obesity[3] |
| Tovorafenib | Treatment of people aged six months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation[3] | |
| Trastuzumab deruxtecan | 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[3] | |
| Vorasidenib | Treatment of people aged twelve years of age and older with grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation, following surgery including biopsy, subtotal resection, or gross total resection[3] | |
| Zanidatamab | Jazz Pharmaceuticals | Treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC3+) biliary tract cancer as detected by an FDA approved test[3] |
| Zenocutuzumab | Treatment of adults with advanced, unresectable or metastatic non-small cell lung cancer harboring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior systemic therapy; and adults with advanced, unresectable or metastatic pancreatic adenocarcinoma harboring an NRG1 gene fusion with disease progression on or after prior systemic therapy[3] |
2023
| Drug | Manufacturer | Indication |
|---|---|---|
| Avacincaptad pegol | To treat geographic atrophy secondary to age-related macular degeneration[5] | |
| Cipaglucosidase alfa | To treat late-onset Pompe disease with miglustat[5] | |
| Dabrafenib | ||
| Dostarlimab | ||
| Eflornithine | ||
| Elranatamab | To treat relapsed or refractory multiple myeloma after at least four lines of therapy[5] | |
| Enfortumab vedotin | ||
| Iptacopan | To treat paroxysmal nocturnal hemoglobinuria[5] | |
| Ivosidenib | ||
| Lecanemab | To treat Alzheimer's disease[5] | |
| Miglustat | ||
| Nedosiran | To lower urinary oxalate levels in primary hyperoxaluria type 1 and relatively preserved kidney function[5] | |
| Nirogacestat | To treat desmoid tumors[5] | |
| Pembrolizumab | ||
| Sutimlimab | ||
| Talquetamab | To treat relapsed or refractory multiple myeloma after at least four therapies[5] | |
| Toripalimab | To treat recurrent or metastatic nasopharyngeal carcinoma with or following other therapies[5] | |
| Tucatinib |
