Temporal Concept
FDA Announces New Rare Disease Approval Programme
FDA; rare disease; conditional approval pathway; accelerated approval; biologics; drug review; surrogate endpoints; real-world data; streamlined process
How Past Endpoints 11 Winners Fared During the 2025 Biotech Downturn
Endpoints 11; biotech downturn 2025; biotech winners; Xaira Therapeutics; biotech awards; industry trends
Amylyx Discontinues Rare Disease Program After Relyvrio Fails to Show Benefit Over Placebo
Amylyx; Relyvrio; AMX0035; progressive supranuclear palsy; ALS; clinical trial failure; rare disease program; placebo; discontinuation
Regeneron Reports Phase 3 Success for Cemdisiran in Rare Disease Generalized Myasthenia Gravis
Regeneron; Phase 3 trial; cemdisiran; generalized myasthenia gravis; rare disease; RNA drug; siRNA; Alnylam Pharmaceuticals; U.S. regulatory submission
Argenx’s Vyvgart Shows Positive Phase 3 Results in Seronegative Myasthenia Gravis
Vyvgart; Argenx; Phase 3; myasthenia gravis; seronegative; ADAPT SERON; AChR-Ab; MuSK+; LRP4+; triple seronegative; FDA label expansion
FDA Begins Daily Publication of Drug Adverse Event Data
FDA; FAERS; adverse event reporting; daily publishing; data modernization; drug safety; real-time monitoring; public health
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
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
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