Drug Approval – Page 4

FDA Introduces New Guidance on AI for Drug Approval Submissions in 2025

12 months ago talkbio0Tagged Advice, Artificial Intelligence, Drug Approval, Drug Development, Medical Devices, regulatory decision-making, United States Food and Drug Administration

FDA, AI, drug approval, guidance, regulatory decision-making, medical devices, artificial intelligence, drug development

FDA Advisory Committee Rejects Lexicon’s Zynquista for Type 1 Diabetes and Chronic Kidney Disease

1 year ago talkbio0Tagged Advisory Committees, Chronic Kidney Diseases, Drug Approval, lexicon, United States Food and Drug Administration, Zynquista

FDA, Lexicon Pharmaceuticals, Zynquista, sotagliflozin, type 1 diabetes, chronic kidney disease, FDA advisory committee, drug approval

European Commission’s Reconsideration of Translarna’s Marketing Authorization: A Critical Turning Point for Duchenne Muscular Dystrophy Treatment

1 year ago talkbio0Tagged CHMP, Commission, Conditional, Decision, Drug Approval, European (ethnic group), Multiple Acyl Coenzyme A Dehydrogenase Deficiency, Muscular Dystrophy, Duchenne, regulatory, Translarna

Translarna, Duchenne muscular dystrophy, European Commission, EMA, CHMP, conditional marketing authorization, drug approval, regulatory decisions

FDA Raises Concerns Over Stealth BioTherapeutics’ Ultra-Rare Disease Drug Ahead of Advisory Committee Meeting

1 year ago talkbio0Tagged 3-Methylglutaconic aciduria type 2, Advisory Committees, concerns, Drug Approval, Effectiveness, Elamipretide, Stealth BioTherapeutics, ultra-rare disease, United States Food and Drug Administration

FDA, Stealth BioTherapeutics, elamipretide, Barth syndrome, ultra-rare disease, advisory committee meeting, drug approval, efficacy concerns

FDA Advisory Panel Rejects PD-1 Inhibitors for Gastric and Esophageal Cancers with Low PD-L1 Expression

1 year ago talkbio0Tagged Biological Markers, Drug Approval, Esophageal Neoplasms, Gene Expression, inhibitors, Malignant neoplasm of stomach, Neoplasms, PD-L1, PDCD1 gene, United States Food and Drug Administration

FDA, PD-1 inhibitors, gastric cancer, esophageal cancer, PD-L1 expression, biomarker, oncology, drug approval

FDA Advisory Committee Approves Zevra Therapeutics’ Arimoclomol for Rare Genetic Disease Treatment

1 year ago talkbio0Tagged Advisory Committees, Arimoclomol, Drug Approval, Hereditary Diseases, Niemann-Pick Diseases, United States Food and Drug Administration

Zevra Therapeutics, Arimoclomol, FDA Advisory Committee, Niemann-Pick disease, rare genetic disease, drug approval

FDA Imposes Three-Month Delay on Ascendis’ Hypoparathyroidism Treatment

2 years ago talkbio0Tagged Ascendis Pharma, Drug Approval, Hypoparathyroidism, regulatory hurdle, three-month setback, United States Food and Drug Administration

Ascendis Pharma, hypoparathyroidism, FDA, drug approval, delay, three-month setback, regulatory hurdle, treatment, endocrine disorder.

Uncategorized

How do the 12 FDA-approved gene therapies break down by medical indication

2 years ago talkbio0Tagged Drug Approval, gene therapy, United States Food and Drug Administration

The 12 FDA-approved gene therapies cover a range of medical indications, primarily focusing on genetic disorders, cancers, and hematological conditions. Here’s a breakdown of these therapies by their medical indications: Genetic Disorders: Luxturna (voretigene neparvovec-rzyl): Approved for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy, which leads to vision loss and may […]