Elevidys And The EMBARK Trial: Is Another Advisory Committee Meeting Coming?

Sarepta says another public meeting is not necessary to expand the label for its DMD gene therapy, but the missed primary endpoint may require input from experts outside the FDA, and the situation renews the spotlight on CBER Director Peter Marks and his initial approval decision.

Sarepta
Sarepta will submit an sBLA requesting an expansion of the Elevidys label. • Source: Shutterstock

Another public discussion of Sarepta Therapeutics, Inc.’s gene therapy for Duchenne muscular dystrophy Elevidys may be on the horizon after the product missed the primary endpoint in its confirmatory study.

The company tried to spin the miss as a success, arguing that secondary endpoint data shows that Elevidys (delandistrogene moxeparvovec-rokl)...

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