Ed Silverman , 2025-04-29 10:30:00
File this under “So close, yet so far.”
After several years of struggling with regulatory hurdles to win approval for its rare disease drug, Stealth BioTherapeutics had expected the U.S. Food and Drug Administration to respond on Tuesday to its marketing application.
But late last week, the company received a letter saying there was a delay. Moreover, the agency did not indicate when it may now complete its review for the drug, which is called elamipretide and was developed to combat Barth syndrome. The rare illness, which causes an enlarged heart, muscle weakness and a shortened life expectancy, afflicts nearly 150 people in the U.S.
This article is exclusive to STAT+ subscribers
Unlock this article — plus in-depth analysis, newsletters, premium events, and news alerts.
Already have an account? Log in
To read the rest of this story subscribe to STAT+.
