Sarepta Therapeutics, Inc.’s bid to use external control data to contextualize the clinical efficacy results for SRP-9001 in Duchenne muscular dystrophy fell flat with the US Food and Drug Administration review team that is assessing the gene therapy, but found a receptive audience with some advisory committee members.
External Controls: Sarepta’s DMD Gene Therapy Not In the Same Boat As Zolgensma, US FDA Says
Sarepta conducted study-level and integrated-level comparison analyses of SRP-9001-treated patients and external controls. However, heterogeneous nature of DMD and potentially moderate treatment effect of SRP-9001 distinguish it from Novaritis' spinal muscular atrophy treatment, where natural history data were used to support single-arm trial results, the FDA said.
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