Following an eventful couple of months with Elevidys (delandistrogene moxeparvovec-rokl), Sarepta Therapeutics has announced that the efficacy supplement for its biologics license application (BLA) of the Duchenne muscular dystrophy (DMD) gene therapy has received priority review by the FDA.
As per the 16 February press release, the purpose of the efficacy supplement is to change Elevidys’ accelerated approval to a traditional approval while also expanding the therapy’s label to treat all DMD patients with a confirmed mutation in the DMD gene. The FDA has set a priority review goal date of 21 June 2024 and will not discuss the supplement in an advisory committee meeting.
Elevidys, which was granted approval in June 2023, is currently indicated to treat ambulatory pediatric DMD patients between the ages of four and five years with a confirmed mutation in the DMD gene. The single-dose gene transfer therapy uses a recombinant adeno-associated virus vector serotype 74 (AAV74) to deliver a microdystrophin transgene that encodes a dystrophin protein.
The priority review comes almost four months after Sarepta announced that the Phase III EMBARK study (NCT05096221) of Elevidys failed to meet its primary endpoint of improvement in motor function as determined by the North Star Ambulatory Assessment (NSAA). The study reported that patients treated with Elevidys improved by 2.6 points on total NSAA score at 52 weeks while patients administered a placebo improved by 1.9 points.
Sarepta was quick to highlight that though the trial missed its primary endpoint, the treatment met all the secondary endpoints.
Bringing Elevidys to market is a joint effort between Sarepta and Roche, with Sarepta being responsible for approval and commercialisation in the US and Roche spearheading approvals outside the US.
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A GlobalData consensus expects Elevidys to pocket $5.7bn in sales in 2029. GlobalData is the parent company of Pharmaceutical Technology.
Sarepta and Roche are not the only companies making strides in the DMD scene. Also in the pivotal stage is Pfizer’s gene therapy fordadistrogene movaparvovec, which is currently being evaluated in the Phase III CIFFREO study (NCT04281485). Elsewhere, Solid Biosciences is in the race with its gene therapy SGT-003, which is currently being evaluated in a Phase I/II study (NCT06138639) and was fast-tracked by the FDA in December 2023.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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