Tiny biotech says its troubles make case for ultra-rare FDA pathway

The struggles of a tiny biotech, Stealth, to develop a therapy for Barth syndrome, a fatal disease that affects about 150 Americans, highlight the regulatory and financial challenges of developing medicines to help patients who have extremely uncommon, serious diseases. These challenges include limited and imperfect data, shifting FDA requirements, and the agency’s inability or unwillingness to apply flexibility that has been exercised for other rare disease treatments.

These difficulties are layered on top of the struggle to create a business model for earning a return on a product when the total possible market is in the 10s or 100s of patients. ...