Sarepta Therapeutics, a Cambridge, Massachusetts-based genetics medicines company, has announced a strategic restructuring plan, which would result in eliminating approximately one-third of its workforce.  

Sarepta has initiated immediate changes to reduce operating expenses and align its cost structure with strategic priorities, aiming to enhance financial flexibility and meet its 2027 financial obligations. Collectively, these measures are projected to deliver approximately $400 million in annual cost reductions, significantly lowering the company’s average annual non- R&D and selling, general, and administrative expenses to between $800 million and $900 million starting in 2026. 

These include: 

• A 36% workforce reduction, impacting approximately 500 employees, projected to generate approximately $120 million in annual cash cost savings in 2026; 

• Pipeline reprioritization expected to deliver approximately $300 million in annual non-personnel cost savings starting in 2026; and  

• Over $100 million in cost savings anticipated through the end of 2025, net of estimated severance and one-time charges totaling $32 million to $37 million 

Sarepta and its partner, Roche, saw a recent setback with Elevidys (delandistrogene moxeparvovec-rok), an AAV vector-based gene therapy, which was nearing blockbuster status with 2024 sales of $821 million. Roche formed a licensing pact in 2019 with Sarepta to develop Elevidys, worth up to $2.85 billion (an upfront payment and equity investment of $1.15 billion, up to $1.7 billion in regulatory and sales milestones, plus royalties on net sales). 

The gene therapy is approved for treating Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscle. The therapy is approved for treating DMD in patients who are ambulatory or non-ambulatory with specified confirmed DMD gene mutations.  

Last month (June 2025), Sarepta provided a safety update following a second reported case of acute liver failure resulting in death in non-ambulatory individuals. As a result, the company is developing an enhanced immunosuppressive regimen in consultation with a panel of multi-disciplinary clinical experts and engaging with regulators. In addition, Sarepta temporarily suspended shipments of Elevidys for non-ambulatory patients while the enhanced immunosuppressive regimen is being evaluated, discussed with regulatory bodies, and put in place. The company is also voluntarily pausing dosing in its Envision clinical trial evaluating Elevidys in older ambulatory and non-ambulatory individuals. That trial also serves as the confirmatory trial required under the FDA’s accelerated approval pathway for non-ambulatory patients. The company says the pause will allow for the evaluation of a protocol amendment to incorporate an enhanced immunosuppressive regimen for the non-ambulatory patient cohort and incorporate any additional feedback from FDA.  

Consistent with other AAV-delivered gene therapies, the FDA has requested that the label include a black box warning for acute liver injury (ALI) and acute liver failure (ALF). Sarepta says it agrees with this change, which appears to resolve any material issues with the ambulant portion of the Elevidys label. 

Source: Sarepta Therapeutics