STAT+: Amid FDA chaos, approval of a rare disease drug gets delayed — again
Stealth BioTherapeutics had expected the FDA to respond on Tuesday to its marketing application for elamipretide to treat Barth syndrome.
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.
