Sarepta Therapeutics crisis is huge blow to Duchenne families, company

Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast The Readout Loud and author of the newsletter Adam’s Biotech Scorecard. You can reach Adam on Signal at stataf.54.

Sarepta Therapeutics confronted one of the most serious crises in its history on Friday, rebuffing a call from the Food and Drug Administration to halt all shipments of its gene therapy for Duchenne muscular dystrophy over safety issues, even as patients and investors expressed growing concern about the company’s decision-making.

The tumult — following a series of patient deaths — has called into question the future of a treatment, called Elevidys, that had been a source of immense hope for Duchenne patients and their families.

Two teenagers who were administered the gene therapy died earlier this year, while a 51-year-old man who received a similar Sarepta gene therapy in a clinical trial for a different form of muscular dystrophy also died. The third death occurred one month ago but only came to light on Thursday evening in media reports.