For a third time, Sarepta Therapeutics has convinced a top Food and Drug Administration official to overrule the prevailing view of their staff and approve a drug for Duchenne muscular dystrophy.
On Thursday evening, the FDA announced it expanded the approval of Elevidys, Sarepta’s Duchenne gene therapy, to cover nearly all patients, regardless of age or wheelchair status, despite the fact that the drug failed a large, Phase 3 trial last year.
That decision, documents concurrently released by the agency show, was made almost exclusively by Peter Marks, the agency’s director of the Center for Biologics Evaluation and Research. He overruled three review teams and two top lieutenants, who wrote that the data Sarepta submitted “cast significant uncertainty regarding the benefits of treatment.”
Click this link for the original source of this article.
Author: Jason Mast and Matthew Herper
This content is courtesy of, and owned and copyrighted by, https://www.statnews.com and its author. This content is made available by use of the public RSS feed offered by the host site and is used for educational purposes only. If you are the author or represent the host site and would like this content removed now and in the future, please contact USSANews.com using the email address in the Contact page found in the website menu.