FDA’s acceptance of Stealth NDA could be sign of improved orphan regulation

Stealth’s announcement that FDA has accepted its NDA for elamipretide to treat Barth syndrome has raised expectations among patients and caretakers that a years-long stalemate between the company and CDER’s Division of Cardiology and Nephrology may be resolved. Faced with a series of regulatory setbacks, including the agency’s 2021 refusal to accept an NDA for the therapy, the company had been on the verge of abandoning the indication and with it the hopes of those suffering from a painful, fatal disease.

The reversal of FDA’s refusal to consider Stealth BioTherapeutics Inc.’s application could signal that FDA staff are putting into practice public commitments from FDA Commissioner Rob Califf and Patrizia Cavazzoni, director of the Center for Biologics Evaluation and Research, to find ways to lower barriers to the development and approval of drugs for rare diseases...