3-Methylglutaconic aciduria type 2
FDA Approves Stealth BioTherapeutics’ Forzinity as First Treatment for Barth Syndrome After Rejection and Delay
FDA approval; Stealth BioTherapeutics; Forzinity; elamipretide; Barth syndrome; rare disease; mitochondrial dysfunction; accelerated approval
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
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 Rejects Stealth BioTherapeutics’ Rare Disease Drug but Leaves Door Open for Accelerated Approval
FDA rejection; Stealth BioTherapeutics; elamipretide; Barth syndrome; rare disease; accelerated approval; muscle strength endpoint; phase 2 trial; regulatory delay
FDA decision on Stealth’s Barth syndrome drug delayed, again
United States Food and Drug Administration, 3-Methylglutaconic aciduria type 2, Decision, Elamipretide
FDA Delays Decision on Stealth BioTherapeutics’ Elamipretide for Barth Syndrome Until April 2025
Barth Syndrome, Elamipretide, FDA Decision, Stealth BioTherapeutics, Drug Approval Delay
FDA Advisory Committee Supports Stealth BioTherapeutics’ Elamipretide for Barth Syndrome Despite Data Concerns
Stealth BioTherapeutics, elamipretide, Barth syndrome, FDA advisory committee, ultra-rare disease, unmet medical need
FDA Raises Concerns Over Stealth BioTherapeutics’ Ultra-Rare Disease Drug Ahead of Advisory Committee Meeting
FDA, Stealth BioTherapeutics, elamipretide, Barth syndrome, ultra-rare disease, advisory committee meeting, drug approval, efficacy concerns
FDA Raises Concerns Over Stealth BioTherapeutics’ Ultra-Rare Disease Drug Approval
Stealth BioTherapeutics, ultra-rare disease, FDA approval, Barth syndrome, elamipretide