New warning on Sarepta’s Elevidys gene therapy for DMD

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.

New warnings and restrictions placed on Sarepta Therapeutics’ gene therapy last week by the Food and Drug Administration have cast a spotlight on an important, but overlooked heart-safety risk.

“Acute, serious, and life-threatening” cases of heart inflammation called myocarditis and elevations of troponin-I, a protein released by dying heart-muscle cells, have been reported in patients treated with Elevidys, Sarepta’s gene therapy for Duchenne muscular dystrophy, according to its prescribing label updated on Friday. 

A previous version of the Elevidys label noted only that “acute and serious” myocarditis and troponin-I elevations had been observed.