Stealth CRL raises questions about FDA’s implementation of rare disease policies

FDA has issued a complete response letter for elamipretide, sending patients with Barth syndrome, along with the drug’s developer, Stealth Biotherapeutics, careening around another unexpected turn on the regulatory rollercoaster.

Although the CRL provides hope that FDA will grant accelerated approval to elamipretide for Barth, the agency’s requirement that Needham, Mass.-based Stealth Biotherapeutics Inc. resubmit the NDA is baffling, CEO Reenie McCarthy told BioCentury. Even more troubling, she said, is the agency’s suggestion that the therapy’s label exclude newborns — a decision that could put lives at risk, as most infants with the syndrome die before their first birthday...