Right now, children with a rare disease called Barth syndrome are running out of time — and out of the only medication that’s helped keep them alive.

Elamipretide, an experimental drug developed by Stealth BioTherapeutics, has shown promising results in improving heart and muscle function in kids with this ultra-rare genetic condition.

But after more than a decade of development, and despite a panel of experts voting to recommend approval in 2024, the FDA has continued to delay.

Now, families are counting how many vials they have left in the fridge.

This week, two babies with Barth syndrome reportedly died.

The FDA says more data is needed. But experts — including members of Congress — are calling on the agency to use its accelerated approval pathway, especially when no other treatment exists and the condition is so rare that large trials are impossible.

Meanwhile, no additional safety issues have been raised. No new clinical trials have been requested. The only thing standing between these children and continued treatment is bureaucracy.

We are calling on the FDA and Department of Health and Human Services to immediately approve elamipretide through the accelerated approval process.

Congress to push for urgent transparency and action on behalf of families with rare diseases.
 

This is a matter of life or death — and the clock is ticking.