United States Food and Drug Administration
FDA Delays Decisions on Regeneron’s Eylea HD Due to Manufacturing Issues
FDA delay; Regeneron; Eylea HD; manufacturing issues; Novo Nordisk facility; biosimilars; pre-filled syringe; macular oedema; retinal vein occlusion; regulatory approval
Nucleus RadioPharma Appoints Former FDA Commissioner Dr. Stephen Hahn as CEO
Nucleus RadioPharma; Stephen Hahn; FDA Commissioner; CEO appointment; radiopharmaceuticals; cancer therapy; supply chain; biotech leadership
FDA Lifts Hold on Rocket Pharmaceuticals’ Pivotal Gene Therapy Study at Lower Dose
FDA; Rocket Pharmaceuticals; gene therapy; Danon disease; clinical hold; RP-A501; dose adjustment; C3 complement inhibitor; capillary leak syndrome; patient death; Phase II trial
FDA Rejects PTC’s Vatiquinone Application for Friedreich’s Ataxia, Requests Further Studies
FDA rejection; PTC Therapeutics; vatiquinone; Friedreich’s ataxia; complete response letter; drug efficacy; additional clinical studies
FDA Rejection Derails PTC Therapeutics’ Rare Disease Hopeful
PTC Therapeutics; FDA rejection; vatiquinone; Friedreich’s ataxia; rare disease; efficacy concerns; complete response letter; clinical trial failure
FDA Extends PDUFA Date for Regenxbio’s Hunter Syndrome Gene Therapy RGX-121
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
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
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
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
Tonmya; FDA approval; fibromyalgia; Tonix Pharmaceuticals; non-opioid analgesic; cyclobenzaprine HCl sublingual tablets; chronic pain; phase 3 clinical trials