Today, it was announced that Sarepta Therapeutics would reduce its workforce by 36%, which is the cutting of around 500 employees from the organization. This is expected to save the company $120 million annually starting in 2026. This wasn’t the only move that it had made; other items that were done were to reprioritize its pipeline to develop small interfering RNA (siRNA) candidates to treat patients with rare diseases. Some examples of what it intends to develop drugs for are myotonic dystrophy type 1 (DM1), Huntington’s disease (HD), and idiopathic pulmonary fibrosis. Advancing this portion of the pipeline makes sense because it is already generating substantial revenues with several of its phosphorodiamidate morpholino oligomer (PMO) candidates.
The RNA-based PMO candidates being given to patients for the treatment of Duchenne Muscular Dystrophy (DMD) were reported to have brought in net product revenue of $231 million in Q2 of 2025. However, when referring to the revenue generated from the entire DMD treatment program during this most recently reported quarter, it was $513 million. This was thanks to the other portion of this portfolio, which is a gene therapy candidate by the name of ELEVIDYS to treat these neuromuscular disorder patients. This gene therapy generated net product revenue of $282 million during this reported second quarter of 2025.
Speaking of ELEVIDYS, it was revealed on June 16th of 2025 by Reuters that there was a second patient death. This patient, who received this gene therapy, suffered from severe liver failure and ultimately died. With that being said, this patient, along with another prior patient who died, prompted the company to speak to the FDA about it. It received notice that the FDA would have to place a black box warning label for both acute liver injury (ALI) and acute liver failure (ALF). To this end, this only satisfied the ambulatory patients. However, both of the DMD patients that died were non-ambulatory patients. This means that the company is not out of the woods yet with respect to this portion of DMD patients.
What it is in the process of doing is submitting a protocol to the FDA about adding an immunosuppressive regimen with sirolimus for ELEVIDYS specifically for non-ambulatory patients. This isn’t going to be a quick process, though, because it is going to have to be incorporated into cohort 8 of its ENDEAVOR trial (Study SRP-9001-103). The point being is that even if Sarepta ultimately gets its protocol submitted, the FDA would not only have to evaluate it but also agree to it. Only then can the company start selling ELEVIDYS for non-ambulatory DMD patients again. The hope is that it can regain compliance with this portion of patients as well. The reason why is because ELEVIDYS is the first and only approved gene therapy for the treatment of patients with DMD. It might be able to do so with dialogue with this U.S. agency or by some other means.
This restructuring is not ideal, but the company believes that it was necessary to meet is 2027 financial obligations. Another downside to it is the fact that the company had to cut out many other ongoing gene therapy programs. For instance, there were studies for the development of a gene therapy to treat patients with limb-girdle muscular dystrophy (LGMD). However, before announcing this restructuring, it had already completed testing for SRP-9003 for the treatment of these patients. As a matter of fact, it intends to file a Biologics License Application (BLA) for this gene therapy targeting types 2E/R4 in the 2nd half of 2025. It hopes, though, that it can continue many of these programs that it doesn’t intend to fund through some partnerships or collaboration agreements.