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.
You may also be interested in...
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.
Unpredictable DMD Progression Complicates Sarepta’s Gene Therapy Efficacy Claims
As with many rare diseases, a clear natural history would make deciphering the data easier, but for now what Sarepta characterizes as stabilization is seen by FDA reviewers as expected improvement based on previous measures of disease progression at younger ages.
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.