STAT+: The crisis over Sarepta’s Duchenne therapy is a mess. But the lesson is clear

In a matter of a few days last week, Sarepta Therapeutics announced major layoffs, acknowledged the death of another patient receiving one of its gene therapies, and was urged by the Food and Drug Administration to suspend shipments of a marketed product for Duchenne muscular dystrophy, Elevidys. The company refused.

Sarepta’s stock has fallen 30% in the past week and 90% since the beginning of the year, meaning a company that appeared poised to become one of biotech’s largest is shrinking dramatically. But this is not just a story about a company stumbling in its communications with the FDA. It is an object lesson in the risks that come with lowering the barriers for evidence for new medicines — not just once, but serially — and about the incentives doing so creates for biotechnology companies. This is a story of what not to do when developing a breakthrough product.

“Sarepta is causing such a polarised debate because the evidence that the drug is effective isn’t gold standard,” David Grainger, a longtime venture capitalist and the chairman of development at Rivus Pharmaceuticals, wrote on the social media site X.

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