FDA approved elamipretide (SS-31), the first treatment for Barth syndrome

Accelerated approval for a peptide that acts in the mitochondria, in a rare and serious disease.

On 19 September 2025, the FDA granted accelerated approval to elamipretide, known in research as SS-31, for Barth syndrome in patients weighing at least 30 kg. It is a rare mitochondrial disease that mainly affects males, with heart failure, fatigue and exercise intolerance.

Elamipretide binds to the inner part of the mitochondria and improves their structure and function. Approval was based on improved strength of the knee-extensor muscle, and the FDA required a later controlled trial to confirm clinical benefit.

In three points

First approved treatment for Barth syndrome
A peptide that acts directly in the mitochondria
Accelerated approval with a confirmatory trial pending

Compounds mentioned

SS-31 elamipretide

Original source: FDA Grants Accelerated Approval to First Treatment for Barth Syndrome
FDA · U.S. Food and Drug Administration

Plain-language summary by SYNTRA LABS based on the original publication and checked against its source. It is not medical advice or a recommendation for use. SYNTRA LABS compounds are for research use only and not for human consumption.