FDA Rejection of Rare Disease Drug Sparks Debate on Approval Pathways
TL;DR Summary
Stealth BioTherapeutics' drug for Barth syndrome, a rare illness affecting up to 150 people in the U.S., was unexpectedly rejected by the FDA, delaying access for patients and straining the company's finances, despite prior advisory committee support and ongoing discussions about new approval pathways.
- A rare disease drug is rejected, even as the FDA talks about new approval pathway statnews.com
- Stealth BioTherapeutics Announces "Path Forward" Despite Disappointing Delay for Ultra-rare Barth Syndrome PR Newswire
- FDA rejects Stealth’s rare disease drug, but offers potential for accelerated approval Endpoints News
- Stealth hit by FDA rejection to twice-delayed filing as it lays off staff Fierce Biotech
- Barth Syndrome Foundation Responds to FDA's Proposed 'Path Forward' for Elamipretide Yahoo Finance
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