Last week Sarepta Therapeutics had a string of bad news relating to its gene therapy ELEVIDYS, which was approved by the FDA to treat patients with ambulatory Duchenne Muscular Dystrophy (DMD). The first batch of bad news came when, on July 21, 2025, it was announced that it would voluntarily pause all shipments of this gene therapy for the treatment of ambulatory DMD patients starting on July 22, 2025. Part of the reason may have been that it faced pressure from the U.S. agency to do this, but the company noted that it wanted to foster a good relationship with the FDA and address any safety concerns that it may have had with respect to it.
If that wasn’t bad enough, its European partner for ELEVIDYS, Roche, revealed that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative recommendation on approval for this drug for the treatment of ambulatory DMD patients ages 3 to 7 for the European Union (EU). Again, it is important to state that this negative opinion wasn’t on the issue in terms of the safety of this gene therapy. Instead, it was in terms of a trial known as EMBARK not achieving the intended primary endpoint over a 1-year period. Both companies are banking on the possibility that there might still actually be a path forward for getting ELEVIDYS approved in the EU. This would be a totality of data from several long-term studies, showing that patients who took it achieved improvements in motor function.
Regardless, Roche is not giving up on its endeavor to get ELEVIDYS approved for this pool of ambulatory DMD patients. Plus, it must be accounted for that this was only a negative opinion of the CHMP. The final decision on whether or not this ends up in the European market remains with the European Commission (EC) to make the final determination of approval. The hope for patients in this territory is that it is ultimately approved, because it is the only gene therapy that goes after the root cause of DMD. An approval in the EU would go a long way to help a large group of these ambulatory patients.
With these two negative events last week, it’s safe to say that things looked very bleak for the future of Sarepta. Until today, when the FDA completely altered course on the halting of shipments for ELEVIDYS for the treatment of patients with ambulatory DMD. Instead, it stated that Sarepta should resume shipments of ELEVIDYS in the targeting of ambulatory DMD patients. The reason why the U.S. agency wanted the pause in the first place was because of an incident involving an 8-year-old in Brazil who died while taking ELEVIDYS. However, Brazilian authorities noted that the death was unrelated to this gene therapy. From there, the FDA concluded the very same thing, and that is why it gave the go-ahead to again resume shipments for this specific group of DMD patients.
This is definitely good news and should be applauded, as this therapy helps a lot of these DMD patients. Unfortunately, the company is not off the hook yet, so to say, when it comes to being able to market its gene therapy for non-ambulatory DMD patients. With the company being able to work well with the FDA in terms of the issues surrounding ambulatory DMD patients, then it might be plausible that it might find a path forward to get it approved to target the other portion of DMD patients. The plan to accomplish this move is to add an immunosuppressive regimen to ELEVIDYS in the hopes that it will stem the problem arising in targeting this group of patients.
That is, to reduce the incidents of acute liver failure for non-ambulatory DMD patients who take this gene therapy. It is believed that the addition of this regimen could prevent higher than expected elevated levels of liver enzymes. If this safety issue can be shown to be resolved in the ENVISION study, then it is quite possible that the company might be able to convince the FDA to get ELEVIDYS on the market to help treat non-ambulatory DMD patients. Speaking of getting permission from the FDA, it needs to do so in order to incorporate sirolimus as an adequate immunosuppressive regimen with ELEVIDYS.
Besides all of these events that occurred, there is another side of its pipeline it intends to focus on. This involves the development of small interfering RNA (siRNA) candidates to target large market indications like myotonic dystrophy type 1 (DM1), idiopathic pulmonary fibrosis (IPF), and Huntington’s disease (HD). In essence, it intends to reveal data from several early-stage trials targeting such indications in the 2nd half of 2025. Should data from these trials turn out to be positive, then there would be other developments that could reignite shareholder value going forward.
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