United States Food and Drug Administration – Page 21

FDA Extends PDUFA Date for Regenxbio’s Hunter Syndrome Gene Therapy RGX-121

4 months ago talkbio0Tagged clemidsogene lanparvovec, Deferred, gene therapy, IDS gene, Infrequent, Mucopolysaccharidosis II, PDUFA, Regenxbio, RGX-121, United States Food and Drug Administration

FDA delay; PDUFA extension; Regenxbio; Hunter syndrome; clemidsogene lanparvovec; RGX-121; gene therapy; Mucopolysaccharidosis II; IDS gene; rare disease

Stealth BioTherapeutics Resubmits Third FDA Application for Rare Disease Drug

4 months ago talkbio0Tagged 3-Methylglutaconic aciduria type 2, access program, Drug Approval, Elamipretide, Infrequent, resubmission, Stealth BioTherapeutics, United States Food and Drug Administration

Stealth BioTherapeutics; FDA resubmission; rare disease; Barth syndrome; elamipretide; drug approval; expanded access program

Federal Appeals Court Overturns FDA Rejection of Vanda’s Hetlioz for Jet Lag Disorder

4 months ago talkbio0Tagged Clinical Trials, Evidence, Federal Appeals Court, Hetlioz, jet lag disorder, New Drug Application, regulatory law, sNDA, United States Food and Drug Administration, Vanda Pharmaceuticals

Vanda Pharmaceuticals; Hetlioz; FDA; jet lag disorder; Federal Appeals Court; supplemental New Drug Application (sNDA); regulatory law; clinical trial evidence

Conflicts of Interest Among Federal Vaccine Advisors at Historic Lows Through 2024

4 months ago talkbio0Tagged ACIP, Advisory Committees, Centers for Disease Control and Prevention (U.S.), JAMA study, Robert F. Kennedy Jr., United States Food and Drug Administration, Vaccines, VRBPAC

conflicts of interest; vaccine advisory committees; ACIP; VRBPAC; CDC; FDA; transparency; Robert F. Kennedy Jr.; JAMA study

FDA Approves Tonix’s Tonmya: First New Fibromyalgia Drug in Over 15 Years

4 months ago talkbio0Tagged Analgesics, Non-Narcotic, Approved, Chronic pain, Clinical Trials, cyclobenzaprine, Fibromyalgia, Hairy Cell Leukemia, Phase 3, sublingual tablets, Tonix, Tonmya, United States Food and Drug Administration

Tonmya; FDA approval; fibromyalgia; Tonix Pharmaceuticals; non-opioid analgesic; cyclobenzaprine HCl sublingual tablets; chronic pain; phase 3 clinical trials

FDA Grants Wegovy 3rd Indication: First GLP-1 Approved for MASH with Moderate-to-Advanced Liver Scarring

4 months ago talkbio0Tagged Accelerated, Approved, Fibrosis, Liver, MASH, noncirrhotic, Nordisk, Product containing glucagon-like peptide 1 receptor agonist (product), semaglutide, United States Food and Drug Administration, Wegovy

Wegovy; FDA approval; MASH; semaglutide; GLP-1 receptor agonist; liver fibrosis; Novo Nordisk; ESSENCE trial; noncirrhotic; accelerated approval

FDA Delays Draw Contrasting Responses: Neurizon Cites Agency Strain, Omeros Takes Softer Tone

4 months ago talkbio0Tagged agency staffing shortages, Amyotrophic Lateral Sclerosis, delays, narsoplimab, NUZ-001, Omeros Corporation, regulatory review, Study on Hold, Transplantation, United States Food and Drug Administration

FDA delays; Neurizon Therapeutics; Omeros Corporation; regulatory review; NUZ-001; narsoplimab; ALS; transplant drugs; agency staffing shortages; clinical hold

FDA Grants Full Approval to Precigen’s Papzimeos for Rare Disease RRP

4 months ago talkbio0Tagged Approved, Episodic, gene therapy, Immunotherapy, Infrequent, orphan drug designation, Papzimeos, Precigen, Recurrent respiratory papillomatosis, RRP, United States Food and Drug Administration

Precigen; Papzimeos; FDA approval; recurrent respiratory papillomatosis (RRP); gene therapy; immunotherapy; rare disease; Orphan Drug designation

HHS Revives ’90s-Era Task Force to Improve Childhood Vaccine Safety

4 months ago talkbio0Tagged Adverse reactions, Advisory Committees, Centers for Disease Control and Prevention (U.S.), CHARGE Syndrome, Childhood, Childhood Vaccines, Jay Bhattacharya, NIH Mouse, Robert F. Kennedy Jr., United States Food and Drug Administration, Vaccine Safety, Vaccines

HHS; childhood vaccines; vaccine safety; task force; Robert F. Kennedy Jr.; Jay Bhattacharya; NIH; FDA; CDC; vaccine oversight; adverse reactions; childhood immunization schedule

Precigen Receives FDA Approval for First Immunotherapy for HPV-Related Recurrent Respiratory Papillomatosis

4 months ago talkbio0Tagged Adenovirus Vector, Approved, Episodic, gene therapy, human papillomavirus, Identifier, Immunotherapy, Papzimeos, Precigen, Recurrent respiratory papillomatosis, RRP, United States Food and Drug Administration, zopapogene imadenovec-drba

Precigen; FDA approval; Papzimeos; zopapogene imadenovec-drba; HPV; recurrent respiratory papillomatosis (RRP); immunotherapy; gene therapy; adenoviral vector; Breakthrough Therapy designation