Breaking: FDA approves Sarepta’s Duchenne gene therapy for nearly all patients

FDA approves Sarepta's Duchenne gene therapy for nearly all patients

By Jason Mast

KRISTOFFER TRIPPLAAR/SIPA VIA AP

The FDA has approved Elevidys, Sarepta's gene therapy for Duchenne muscular dystrophy, for patients who can walk on their own.

The FDA's decision vastly widens access to the treatment. For the last year, only 4- and 5-year-olds could obtain it, which proved gut-wrenching for families who found themselves just over the line.

The drug will now be fully approved for all patients ages 4 and up who can walk on their own. For patients who rely on wheelchairs, the agency granted accelerated approval, a form of conditional authorization that will have to be confirmed in a larger study.

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