Back to today’s briefing

Story perspectives

FDA Approves First Barth Syndrome Treatment Amid Challenges

9/30/2025

50 10 Full Breakdown

Summary

Story summary

The Food and Drug Administration (FDA) approved elamipretide as the first treatment for Barth syndrome.
Elamipretide improves mitochondrial function and showed efficacy in clinical trials.
The approval process faced delays and challenges, including FDA rejections and Stealth BioTherapeutics’ financial issues.
Evinacumab-dgnb was approved for homozygous familial hypercholesterolemia in children as young as one year old.

1 of 1

Story summary

The Food and Drug Administration (FDA) approved elamipretide as the first treatment for Barth syndrome.
Elamipretide improves mitochondrial function and showed efficacy in clinical trials.
The approval process faced delays and challenges, including FDA rejections and Stealth BioTherapeutics’ financial issues.
Evinacumab-dgnb was approved for homozygous familial hypercholesterolemia in children as young as one year old.