Approval beckons for Sarepta’s gene therapy

Sarepta has shown again that a lack of positive placebo-controlled data need not be a barrier for regulatory approval. The group’s Duchenne muscular dystrophy (DMD) gene therapy, SRP-9001, looks set to get the thumbs-up after a narrow panel nod last Friday, despite scathing FDA briefing documents. One issue raised was that receiving SRP-9001, delivered via adeno-associated viral vectors, would preclude patients from later receiving another AAV-based therapy owing to immunogenicity. But there is not much else in the pipeline: Sarepta’s big gene therapy rival is Pfizer, whose PF-06939926 was previously put on hold after a patient death. Another competitor, Solid Biosciences, abandoned its lead project, SGT-001, in favour of the next-gen SGT-003, which is not yet in the clinic. Unlike those for other Sarepta projects, at least SRP-9001’s expected accelerated approval is set to be followed fairly swiftly with confirmatory data from the Embark trial, due in the fourth quarter. The Roche-partnered asset could bring in nearly $3bn by 2028, according to sellside consensus compiled by Evaluate Pharma. The other big event in DMD this year will be phase 3 data on Fibrogen’s pamrevlumab, which have become increasingly important for that company following a recent miss with roxadustat.