Things were already bad for Sarepta Therapeutics with respect to its gene therapy ELEVIDYS for the treatment of patients with Duchenne muscular dystrophy. In March of 2025 it noted a death for one patient who died of acute liver failure (ALF), and then later on in June of 2025 a second patient had died due to the same cause. Both of these patients were non-ambulatory DMD patients. At that time, the company had only halted shipments of this gene therapy to non-ambulatory patients because of these deaths. Well, things took a huge turn this past week, as on Monday, July 21, 2025, the company provided an update that it would voluntarily pause all U.S. shipments of ELEVIDYS for DMD patients. The effective date for shipments to be paused was set to be Tuesday, July 22, 2025.
It believed this was a necessary step to foster a good relationship with the FDA and get to the bottom of what it could do to eventually bring ELEVIDYS back to the U.S. market. Besides the two patient deaths who had non-ambulatory DMD, things were further complicated with a third patient death. However, this time around the patient who had died was a 51-year-old man with non-ambulatory limb-girdle muscular dystrophy (LGMD) who took a single investigational dose of SRP-9004. All of these events are what led to the company having to voluntarily pause all shipments of ELEVIDYS in the United States.
The thing is that this gene therapy was developed by Roche in collaboration with Sarepta Therapeutics. Fast forward to July 25, 2025, and further woes were brought upon both companies. That’s because the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion with respect to granting marketing approval of ELEVIDYS for ambulatory patients ages 3 to 7 with DMD in the European Union. It is important to note, though, that the negative opinion given was not on the basis of safety. Instead, the regulatory body had a concern that the phase 3 EMBARK study didn’t meet the primary endpoint within a 1-year period.
However, Roche touted the fact that this gene therapy performed well in terms of secondary endpoints dealing with functional outcomes for patients when being compared to that of placebo. The bottom line is that Roche is not giving up, and it believes it might be able to work with the EMA to get ELEVIDYS out to ambulatory DMD in the EU. The hope is that there is a way to get this gene therapy out to these specific DMD patients. The reason why is because it is the first and only of its kind to be approved to target the root cause of this disease. Roche is a big pharma company and is not heavily affected by the setback of this gene therapy. On the other hand, Sarepta is already likely to see declining revenues in the coming quarters because of its setback in the United States.
The collaboration agreement was formed back in 2019, by which Sarepta granted Roche rights to develop, manufacture, and commercialize ELEVIDYS in global territories outside of the United States. While Sarepta retained all rights to this gene therapy for the treatment of these patients with DMD in the United States. The way that this therapy works is that it is set up to deliver a micro-dystrophin to muscle cells in hopes of improving structure and function for these patients. This negative CHMP opinion of ELEVIDYS is another stumble for this embattled gene therapy. With all the issues that have cropped up for it, it remains to be seen if Sarepta can work with the FDA to start delivering the therapy to DMD patients in the United States. Plus, now Roche has to work with the EMA in hopes that it can convince it to give it the authority to market ELEVIDYS for ambulatory DMD patients in the EU.