Some reviewers opposed FDA's approval of new drug for life-threatening disease

When elamipretide, a drug sold by Stealth BioTherapeutics under the brand name Forzinity, was approved by the U.S. Food and Drug Administration (FDA) for the treatment of Barth syndrome, it represented a breakthrough of sorts for modern medicine. New reporting provides additional context about the historic approval.

Elamipretide was the first treatment ever approved for Barth syndrome, an incredibly rare genetic condition linked to heart failure, cardiomyopathy, neutropenia, fatigue and other severe symptoms in young patients, primarily men. Though it is incredibly expensive—nearly $800,000 per year—there is a hope that health insurance could follow in the months ahead, improving access to the drug for many more patients.

After initially declining to approve elamipretide in 2021, the FDA agreed to an accelerated approval in September 2025. The agency even issued a statement to highlight the news. New reporting from Reuters, however, shows that eight reviewers working with the FDA did not feel the drug should have been approved. 

Reuters reviewed several FDA documents, highlighting some of the concerns that were shared during the approval process. Elamipretide was unable to outperform a placebo in one 28-week study, for example, and a separate study failed to show benefits to the new drug over a placebo. 

It’s worth noting, of course, that the FDA’s review team does not always agree on medications. Also, as Reuters mentioned in its own reporting, patient advocates and lawmakers pushed hard for elamipretide to be approved due to the fact that no other treatment options are currently available. 

Stealth BioTherapeutics plans on officially launching elamipretide (Forzinity) in December. 

To learn more, click the link below and read the full breakdown from Reuters: