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

 
• By Derrick Gingery

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) offers a clinically meaningful treatment benefit and the primary endpoint, the North Star Ambulatory Assessment

Key Takeaways

  • Elevidys missed the primary endpoint in the EMBARK trial, but Sarepta points to secondary endpoint data as proof of efficacy in Duchenne muscular dystrophy

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