External Controls: Sarepta’s DMD Gene Therapy Not In the Same Boat As Zolgensma, US FDA Says

17 May 2023

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.

Looking out window — Sarepta looked outside the SRP-9001 clinical program to contextualize the gene therapy's clinical efficacy results. • Source: Shutterstock

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.

At the 12 May meeting of the Cellular, Tissue and Gene Therapies Advisory Committee, agency review staff said DMD is too heterogeneous a disease, and any potential treatment effect with SRP-9001 is too moderate, for

External Controls: Sarepta’s DMD Gene Therapy Not In the Same Boat As Zolgensma, US FDA Says

More from US FDA Performance Tracker

More from Regulatory Trackers