STAT+: What the Sarepta decision means for Duchenne patients, the company, and the FDA
Sarepta Therapeutics always wins. That was a given, even before the Food and Drug Administration expanded approval of the company’s Duchenne muscular dystrophy drug on Thursday. Will boys with Duchenne also benefit? I hope they do, but it’s uncertain. And is this a good look for the FDA? No, it is not.
There is much to unpack from the unilateral decision by Peter Marks, the FDA’s top regulator of gene therapies, to expand the use of Sarepta’s Elevidys to nearly all Duchenne patients. Some thoughts:
Sarepta is unshackled. The broad approval — its best-case scenario — gives the company freedom to sell its gene therapy almost without restrictions. Manufacturing capacity — and how fast it can be upgraded — might be one of the only brakes right now on Sarepta’s effort to hit peak U.S. sales of $2-3 billion.
