An Ultra-Rare Disease Drug Developer Tries to Navigate Regulatory Uncertainty

Reenie McCarthy, CEO of Stealth Biotherapeutics, discusses the challenges the company has faced in seeking FDA approval for its experimental therapy to treat the rare and life-threatening condition Barth syndrome, the lack of consistency the company found from division to division within the agency, and why this could have a chilling effect on the development of ultra-rare disease therapies if left unaddressed.