(Reuters) -The U.S. Food and Drug Administration has delayed its decision on marketing approval for Stealth BioTherapeutics’ treatment for a rare genetic condition that typically affects boys, the drug developer said on Tuesday.
The Needham, Massachusetts-based company’s lead drug, elamipretide, is a potential treatment for Barth syndrome – a condition that affects the heart, muscles, immune system and delays growth.
WHY IT’S IMPORTANT
This marks the second delay for the review of elamipretide, as the health regulator in January had said it required more time to evaluate additional data submitted by the company as part of its application.
Currently, there are no FDA-approved therapies for the treatment of Barth syndrome.
CONTEXT
The latest delay comes during a period of mass layoffs, including the ouster of high-ranking scientists, at the FDA as part of a major overhaul under Health Secretary Robert F. Kennedy Jr. The changes have prompted concerns that regulatory review of treatments could be disrupted.
KEY QUOTE
“We hope to gain more information on the revised action date in the coming days,” Stealth CEO Reenie McCarthy said. The agency typically provides a new action date in case of delayed decisions.
The FDA has confirmed post-marketing requirements and had discussions on the labeling of the therapy, the company said.
BY THE NUMBERS
The genetic condition, Barth syndrome, affects fewer than 150 individuals in the United States and less than 300 individuals worldwide, according to the company.
(Reporting by Sriparna Roy in Bengaluru; Editing by Shailesh Kuber)
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