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’s DMD Gene Therapy: Approval Delayed And Indication Narrowed, But Still On Track
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.
Slim Adcomm Majority Boosts Sarepta’s Gene Therapy In Duchenne Muscular Dystrophy
Accelerated approval seems imminent after a majority of the US FDA’s Cellular, Tissue and Gene Therapies Advisory Committee voted the risk-benefit balance supported it.
Sarepta’s DMD Gene Therapy: FDA Says Concluding Effectiveness Or Ineffectiveness Is ‘Challenging’
Concerns about the clinical trials, the manufacturing, and the preclusive nature of Sarepta's gene therapy for Duchenne muscular dystrophy raise questions ahead of the upcoming US FDA advisory committee meeting.