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.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

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

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.