United States Food and Drug Administration – Page 23

Elevidys Sales Surge in Sarepta Q2 2025 Despite Safety Concerns

4 months 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

4 months 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

4 months 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

4 months 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

Scientists Protest FDA’s Rejection of Replimune’s Tumor Destroying Therapy

4 months ago talkbio0Tagged Clinical Trials, Drug Approval, Heterogeneity, IGYNTE, melanoma, Oncolytic Immunotherapy, Rejection, Replimune, RETINITIS PIGMENTOSA 1, scientist protest, United States Food and Drug Administration

Replimune; RP1; FDA rejection; oncolytic immunotherapy; melanoma; clinical trial; IGYNTE study; scientist protest; drug approval; patient heterogeneity

Replimune Awaits FDA Direction After Complete Response Letter for Advanced Melanoma Therapy

4 months ago talkbio0Tagged Accelerated, Advanced Melanoma, Approved, Cancer Therapeutic Procedure, Complete, IGNYTE trial, nivolumab, Replimune, Response Letter, RETINITIS PIGMENTOSA 1, United States Food and Drug Administration, vusolimogene oderparepvec

Replimune; FDA; Complete Response Letter; RP1; vusolimogene oderparepvec; nivolumab; advanced melanoma; cancer treatment; IGNYTE trial; accelerated approval

Untangling Sarepta’s Gene Therapy Fallout and Trust Deficit

4 months ago talkbio0Tagged AAVrh74 vector, clinical holds, ELEVIDYS, gene therapy, Muscular Dystrophies, Limb-Girdle, Muscular Dystrophy, Duchenne, patient deaths, Regulatory Action, United States Food and Drug Administration

Sarepta Therapeutics; gene therapy; Elevidys; Duchenne muscular dystrophy; limb-girdle muscular dystrophy; patient deaths; FDA; AAVrh74 vector; regulatory action; clinical holds; transparency

Novo Nordisk’s Leadership Shakeup and Wider Implications; Ex-FDA Official Takes Regulatory Role at GSK

5 months ago talkbio0Tagged CEO, competition, Drops - Drug Form, drug market, Leadership, Maziar Mike Doustdar, Nordisk, Obesity, Prices, Regulatory Affairs, Shakeup, SmithKline Beecham, United States Food and Drug Administration

Novo Nordisk; leadership shakeup; new CEO; Maziar Mike Doustdar; share price drop; obesity drug market; competition; GSK; regulatory affairs; Former FDA