FDA Raises Concerns Over Stealth BioTherapeutics’ Ultra-Rare Disease Drug Approval

FDA Doubts Approval:
The FDA has expressed doubts that the data submitted by Stealth BioTherapeutics for its ultra-rare disease drug, elamipretide, are sufficient for approval or even accelerated approval.

Barth Syndrome Treatment:
Elamipretide is intended to treat Barth syndrome, a disorder that affects approximately 150 Americans and can cause an enlarged and weakened heart.

Clinical Trial Challenges:
Stealth's CEO has expressed concerns about designing a clinical trial that would meet the FDA's expectations, potentially leading to the abandonment of the drug.

Need for Dedicated Rare Disease Center:
The FDA's current structure is criticized for being inefficient in handling rare disease drug approvals, leading to calls for a dedicated "Center for Rare Diseases" to streamline the process.

Impact on Innovation:
The FDA's inconsistent review process for rare disease drugs can hinder innovation and lead to additional costly trials, emphasizing the need for a more focused approach.