Infrequent
FDA Grants Accelerated Approval to Stealth BioTherapeutics’ Forzinity, the First Treatment for Barth Syndrome
FDA approval; Stealth BioTherapeutics; Forzinity; elamipretide; Barth syndrome; mitochondrial disease; accelerated approval; rare disease; pediatric disease; clinical trial
Regeneron Prepares U.S. Submission for Garetosmab After Breakthrough Phase III Result in Ultra-Rare FOP
Regeneron; garetosmab; FOP; fibrodysplasia ossificans progressiva; Phase III; OPTIMA trial; bone lesion prevention; rare disease; FDA submission; orphan drug
Servier Supercharges Neuro Pipeline with $450M Deal for Fragile X Syndrome Candidate
Servier; Kaerus Bioscience; KER-0193; Fragile X syndrome; neurology pipeline; $450 million deal; orphan drug designation; rare pediatric drug designation; Phase 2 trial; acquisition
FDA Launches New Submission Program for Rare Disease Gene Therapies via CDER and CBER
CDER; CBER; rare disease; gene therapy; FDA; submission program; accelerated approval; RDEP; regulatory guidance; rare genetic disorders
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
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
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 Grants Full Approval to Precigen’s Papzimeos for Rare Disease RRP
Precigen; Papzimeos; FDA approval; recurrent respiratory papillomatosis (RRP); gene therapy; immunotherapy; rare disease; Orphan Drug designation