United States Food and Drug Administration – Page 3

IO Biotech’s cancer vaccine Cylembio narrowly misses Phase III primary endpoint in advanced melanoma, but company eyes FDA filing in 2025

2 weeks ago talkbio0Tagged biotech, Blackfoot language, Clinical Trials, Cylembio, Drops - Drug Form, etimupepimut, Hazard Ratio, Io satellite, Keytruda, melanoma, Overall Survival, P-Value, pembrolizumab, Phase 3, Progression-Free Survival, United States Food and Drug Administration

Cylembio; IO Biotech; melanoma; Phase 3; pivotal trial; progression-free survival (PFS); hazard ratio 0.77; p-value 0.056; Keytruda (pembrolizumab); FDA BLA; overall survival (OS) trend; IOB-013/KN-D18; NCT05155254; imsapepimut; etimupepimut; stock drop

Boehringer Ingelheim breaks into oncology as FDA grants accelerated approval to Hernexeos (zongertinib) for HER2 TKD–mutated NSCLC

2 weeks ago talkbio0Tagged Accelerated, Approved, Beamion, Boehringer Ingelheim, competition, Confirmatory Trial, Enhertu, erbB-2 Receptor, Frequency of Responses, HER2-mutant, Hernexeos, Identifier, LUNG-1, Mutation, Non-Small Cell Lung Carcinoma, non-squamous NSCLC, Oncomine Dx Target Test, oral targeted therapy, Priority Review, Requirement, TKD, United States Food and Drug Administration, Zongertinib

Boehringer Ingelheim; Hernexeos; zongertinib; FDA approval; accelerated approval; non-squamous NSCLC; HER2 TKD mutations; Oncomine Dx Target Test; Beamion LUNG-1; objective response rate; first oral targeted therapy for HER2-mutant NSCLC; priority review; breakthrough therapy designation; confirmatory trial requirement; competition with Enhertu

FDA restricts bluebird bio’s Skysona to CALD patients without suitable stem cell donors after higher blood cancer risk emerges

2 weeks ago talkbio0Tagged boxed, CALD, Cautionary Warning, Cerebral Adrenoleukodystrophy, Donor person, Elivaldogene autotemcel, gene therapy, Hematologic Neoplasms, HLA Match, label update, Leukemia, Myelocytic, Acute, MYELODYSPLASTIC SYNDROME, Safety, Skysona, United States Food and Drug Administration

FDA; bluebird bio; Skysona; elivaldogene autotemcel; eli-cel; cerebral adrenoleukodystrophy; CALD; hematologic malignancies; myelodysplastic syndrome (MDS); acute myeloid leukemia (AML); boxed warning; label update; postmarketing safety; HLA-matched donor; gene therapy safety

FDA tightens Skysona label, restricting use to CALD patients without alternative options due to increased blood cancer risk

2 weeks ago talkbio0Tagged boxed, CALD, Cautionary Warning, Cerebral Adrenoleukodystrophy, Elivaldogene autotemcel, gene therapy, Hematologic Neoplasms, HLA‑matched donor, labeling changes, Leukemia, Myelocytic, Acute, MYELODYSPLASTIC SYNDROME, Safety, Skysona, United States Food and Drug Administration

FDA; bluebird bio; Skysona; elivaldogene autotemcel; labeling changes; boxed warning; hematologic malignancy; myelodysplastic syndrome; acute myeloid leukemia; cerebral adrenoleukodystrophy; CALD; HLA‑matched donor; gene therapy safety; post‑marketing reports

AbbVie and Genmab Move to Expand Use of T Cell Engager Epkinly Following Strong Phase III Results

2 weeks ago talkbio0Tagged AbbVie, Antibodies, Bispecific, Approved, Epcoritamab, EPKINLY, expansion, Genmab, Labels (device), lenalidomide, Lymphoma, Follicular, Phase 3 Clinical Trials, rituximab, T-Cell engager, United States Food and Drug Administration

Epkinly; epcoritamab; AbbVie; Genmab; follicular lymphoma; T cell engager; bispecific antibody; Phase III trial; FDA approval; label expansion; Rituximab; Lenalidomide

FDA Launches PreCheck Program to Accelerate Domestic Drug Manufacturing

2 weeks ago talkbio0Tagged alpha 1-antitrypsin, Construction worker, domestic drug manufacturing, Facility (object), National Security, Pharmaceutical Supply Chain, PreCheck, regulatory streamlining, Trump administration, United States Food and Drug Administration

FDA PreCheck; domestic drug manufacturing; pharmaceutical supply chain; API; regulatory streamlining; facility construction; national security; Trump administration

Elevidys Sales Surge in Sarepta Q2 2025 Despite Safety Concerns

2 weeks ago talkbio0Tagged ELEVIDYS, gene therapy, Pauses, Performance, Safety Issues, Sales - occupational activity, United States Food and Drug Administration

Sarepta Therapeutics; Elevidys; Q2 2025; gene therapy; sales performance; FDA; safety issues; shipment pause

Sarepta Pursues Efficiency Measures Amid $1 Billion Debt Due in 2027

2 weeks ago talkbio0Tagged 2025, debt, decline, Efficiency, ELEVIDYS, Financial cost, financial results, Financial savings, Measures, Muscular Dystrophy, Duchenne, restructuring, United States Food and Drug Administration

Sarepta Therapeutics; debt 2027; efficiency measures; ELEVIDYS; cost savings; restructuring; financial results 2025; FDA; Duchenne muscular dystrophy; stock decline

FDA Grants Accelerated Approval to Jazz’s Dordaviprone (Modeyso), the First Treatment for Ultra-Rare Brain Tumor

2 weeks ago talkbio0Tagged Accelerated, Approved, Brain Neoplasms, Chimerix acquisition, Confirmatory Trial, Dordaviprone, midline glioma, Modeyso, pediatric brain cancer, United States Food and Drug Administration

Dordaviprone; Modeyso; FDA approval; H3 K27M-mutant diffuse midline glioma; ultra-rare brain tumor; Jazz Pharmaceuticals; accelerated approval; pediatric brain cancer; Chimerix acquisition; confirmatory trial

Vertex Drops Next-Generation Pain Drug After Phase 2 Failure, Casts Doubt on Broader Journavx Expansion

3 weeks ago talkbio0Tagged Acute onset pain, Approved, Drug Development, Journavx, non-opioid pain drugs, Pain management, Peripheral neuropathic pain, Sodium Channel Blockers, United States Food and Drug Administration, VX-993

Vertex Pharmaceuticals; Journavx; VX-993; pain management; acute pain; phase 2 clinical trial; sodium channel inhibitor; non-opioid pain drugs; FDA approval; peripheral neuropathic pain; drug development