Sarepta Therapeutics at all times wins. That was a given, even earlier than the Meals and Drug Administration expanded approval of the corporate’s Duchenne muscular dystrophy drug on Thursday. Will boys with Duchenne additionally profit? I hope they do, nevertheless it’s unsure. And is that this search for the FDA? No, it isn’t.
There may be a lot to unpack from the unilateral determination by Peter Marks, the FDA’s prime regulator of gene therapies, to develop the usage of Sarepta’s Elevidys to almost all Duchenne sufferers. Some ideas:
Sarepta is unshackled. The broad approval — its best-case situation — offers the corporate freedom to promote its gene remedy virtually with out restrictions. Manufacturing capability — and how briskly it may be upgraded — could be one of many solely brakes proper now on Sarepta’s effort to hit peak U.S. gross sales of $2-3 billion.
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