Sarepta’s DMD Gene Therapy, Like Exondys 51, Is Foundational, Advocates Argue
The Duchenne muscular dystrophy candidate should be approved because it will help grow development in the space, patient groups argue, not just because of the value the gene therapy could provide to those receiving the treatment itself.
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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 Plots Multi-Study Label Expansion For Elevidys To Eventually Cover Nearly All DMD Population
Studies supporting label expansion already are under way, with more targeting those currently ineligible, including boys with pre-existing antibodies, set to begin later this year, after the initial accelerated approval only allowed use in four- and five-year old patients.
As Sarepta again brings a product application from the brink of rejection to the cusp of approval, the one-month review delay creates more assurance the confirmatory EMBARK trial will be completed as well as providing more time for label negotiations with the US FDA.