FDA Grants Accelerated Approval to Stealth BioTherapeutics’ Forzinity, the First Treatment for Barth Syndrome on September 24, 2025 3-Methylglutaconic aciduria type 2 Accelerated Approved Elamipretide Forzinity Infrequent Mitochondrial Diseases Pediatric Disorder Stealth BioTherapeutics United States Food and Drug Administration +
FDA Approves Stealth BioTherapeutics' Forzinity as First Treatment for Barth Syndrome After Rejection and Delay on September 22, 2025 3-Methylglutaconic aciduria type 2 Abnormality of mitochondrial metabolism Accelerated Approved Elamipretide Forzinity Infrequent Stealth BioTherapeutics United States Food and Drug Administration +
Stealth BioTherapeutics Resubmits Third FDA Application for Rare Disease Drug on August 20, 2025 3-Methylglutaconic aciduria type 2 access program Drug Approval Elamipretide Infrequent resubmission Stealth BioTherapeutics United States Food and Drug Administration +
FDA Rejects Stealth BioTherapeutics' Rare Disease Drug but Leaves Door Open for Accelerated Approval on May 29, 2025 3-Methylglutaconic aciduria type 2 Accelerated Approved Elamipretide Infrequent muscle strength endpoint Phase 2 trial rejection Stealth BioTherapeutics United States Food and Drug Administration +
FDA decision on Stealth's Barth syndrome drug delayed, again on April 30, 2025 3-Methylglutaconic aciduria type 2 Decision Elamipretide United States Food and Drug Administration +