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.
2025 Drug Manufacturer Indication Aficamten The treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms AvutometinibThe treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapyBrensocatibThe treatment of non-cystic fibrosis bronchiectasis (NCFB) in adult and pediatric patients 12 years of age and olderDatopotamab deruxtecanThe 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 therapyDiazoxide cholineThe treatment of hyperphagia in adults and pediatric patients 4 years of age and older with Prader-Willi syndromeDoxecitine/doxribtimineTreatment of thymidine kinase 2 deficiency (TK2d) in adults and pediatric patients with an age of symptom onset on or before 12 yearsDurvalumabIn 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)EpcoritamabIn combination with lenalidomide and rituximab for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL)EsketamineFor treatment-resistant depression (TRD) in adults, as monotherapy or in conjunction with an oral antidepressantEvinacumabAs 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)Trastuzumab deruxtecanThe 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 settingTrastuzumab deruxtecanIn 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 testGemcitabine-Releasing Intravesical SystemTreatment 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 tumorsGlecaprevir/pibrentasvirThe treatment of acute hepatitis C virus (HCV) infection in adults and pediatric patients ages 3 to less than 18 years of ageInebilizumabFor use of in adult patients with immunoglobulin G4- Related Disease (IgG4-RD)IpilimumabIn 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)IptacopanThe treatment of adults with complement 3 glomerulopathy (C3G), to reduce proteinuriaLenacapavirPre-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 acquisitionNarsoplimabTreatment of adult and pediatric patients two years of age and older with hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA)NerandomilastThe treatment of idiopathic pulmonary fibrosis in adult patientsNerandomilastThe treatment of progressive pulmonary fibrosis in adult patientsNivolumabIn 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)ObinutuzumabTreatment 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)PenpulimabPenpulimab-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 therapyPlozasiranAs an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS)SemaglutideFor 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 adultsSevabertinibTreatment 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 therapySibeprenlimabTo reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk for disease progressionSotorasibIn 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 chemotherapySunvozertinibThe 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 chemotherapySuzetrigineThe treatment of moderate to severe acute pain in adultsTaletrectinibThe treatment of adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC)Telisotuzumab vedotinThe 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 therapyZiftomenibTreatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation who have no satisfactory alternative treatment optionsZongertinibTreatment 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
2024