Posted by Michael Wonder on 22 Sep 2016
The FDA did the right thing by approving a novel drug for a disabling disease
22 September 2016 - The FDA’s leadership did the right thing, according to Elaine Schattner in Forbes magazine.
For months, the FDA weighed on eteplirsen, a new molecular medicine designed to treat patients with Duchenne muscular dystrophy, a severe and disabling condition affecting children. This week, the agency said yes.
The medicine, developed by Sarepta Therapeutics, branded Exondys 51, received accelerated approval. Many affected patients, and investors, cheered this news.
The FDA’s decision was controversial. Some, though not all, scientists and doctors who evaluated eteplirsen raised concerns and said it shouldn’t be approved, mainly for the lack of hard evidence that it works. Last spring, an advisory panel to the FDA voted against approval. There has been no placebo-controlled trial of this targeted drug, and it’s likely there never will be.
