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.

The late-stage DMD pipeline
Project Company Description Note
SRP-9001 (delandistrogene moxeparvovec) Sarepta/Roche Microdystrophin gene therapy Adcom voted 8-6 in favour of accelerated approval; Pdufa 29 May 2023; interim Embark data Q4 2023, full data "early 2024"
Vamorolone Santhera Pharmaceuticals Mineralocorticoid receptor antagonist Pdufa 26 Oct 2023
Phase 3
Givinostat Italfarmaco HDAC inhibitor Ph3 Epidys data reported Jun 2022
Pamrevlumab Fibrogen Anti-CTGF antibody Lelantos-1 non-ambulatory, data due Q2 2023; Lelantos-2, ambulatory, data due Q3 0223
CAP-1002 Capricor Therapeutics Allogeneic cardiosphere-derived cell therapy Hope-3 interim data due Q4 2023
PF-06939926 (fordadistrogene movaparvovec) Pfizer Microdystrophin gene therapy Ciffreo data due by H1 2024; 2yr ph1b data at ASGCT 2023
TAS-205 Otsuka Haematopoietic prostaglandin D synthase inhibitor Reach DMD, Japan only, completes May 2027
Source: Evaluate Pharma &

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