Story perspectives

FDA Approves First Treatment for Barth Syndrome: Forzinity

9/20/2025

37 6

Summary

Story summary

The FDA approved Forzinity (elamipretide) for Barth syndrome on September 19, 2025, making it the first treatment for this rare mitochondrial disease.
Barth syndrome mainly affects males, causing severe heart failure in infancy and lower life expectancy.
Forzinity enhances knee muscle strength, potentially improving patients' mobility.
The approval followed previous rejections and a lengthy review process, with a post-approval trial mandated to confirm benefits.
Stealth Biotherapeutics experienced financial strain and staff layoffs due to the extended approval timeline.

1 of 1

Story summary

The FDA approved Forzinity (elamipretide) for Barth syndrome on September 19, 2025, making it the first treatment for this rare mitochondrial disease.
Barth syndrome mainly affects males, causing severe heart failure in infancy and lower life expectancy.
Forzinity enhances knee muscle strength, potentially improving patients' mobility.
The approval followed previous rejections and a lengthy review process, with a post-approval trial mandated to confirm benefits.
Stealth Biotherapeutics experienced financial strain and staff layoffs due to the extended approval timeline.