FDA Raises Concerns Over Stealth BioTherapeutics’ Ultra-Rare Disease Drug Ahead of Advisory Committee Meeting

Drug and Disease:
Stealth BioTherapeutics' drug, elamipretide, is intended to treat Barth syndrome, a progressive and fatal mitochondrial disease.

FDA Concerns:
The FDA has raised doubts about the sufficiency of the data submitted by Stealth BioTherapeutics for approval, citing that the drug missed its primary efficacy endpoints in the main study.

Advisory Committee Meeting:
An advisory committee meeting is scheduled to discuss the drug's efficacy and whether the data supports its approval.

Rare Disease Challenges:
Developing drugs for rare diseases like Barth syndrome is challenging due to the small patient population and complex biology, which can make conducting clinical trials difficult.

Regulatory Context:
The FDA works to support the development of drugs for rare diseases through various incentives and programs, including the Orphan Drug Act, which provides tax credits, fee waivers, and potential market exclusivity.