FDA Delays Decision on Stealth BioTherapeutics’ Elamipretide for Barth Syndrome Until April 2025

FDA Decision Delayed:
The FDA has delayed its decision on Stealth BioTherapeutics' New Drug Application (NDA) for elamipretide, a potential treatment for Barth syndrome, until April 29, 202545.

Reason for Delay:
The delay is due to the FDA needing more time to review "major amendments" submitted by Stealth BioTherapeutics in response to recent information requests5.

Previous Developments:
Stealth BioTherapeutics initially submitted the NDA in August 2021, but it was refused by the FDA. The company later received a pre-NDA meeting to discuss new data collected during the Open Label Extension (OLE) period13.

Advisory Committee Recommendation:
An FDA advisory committee recommended approval of elamipretide for the treatment of Barth syndrome in September 2024, with a 10-6 vote23.

Community Advocacy:
The Barth Syndrome Foundation (BSF) and other patient advocacy groups have been actively advocating for the approval of elamipretide, emphasizing the need for treatments for this ultra-rare and life-threatening disease13.

Sources:

1. https://www.barthsyndrome.org/newsevents/pressreleases.html

2. https://umdf.org/umdf-reacts-to-fda-advisory-committees-elamipretide-recommendation/

3. https://barthsyndrome.org/newsevents/newsarchives.html

4. https://www.fiercebiotech.com/biotech/fda-delays-decision-stealths-2nd-shot-approval-ultra-rare-disease-med

5. https://c.firstwordpharma.com/firstwordgroup/assets/images/fdadelay.jpg?sa=X&ved=2ahUKEwil38TmuY6LAxWr38kDHfDlMAoQB16BAgEEAI