Sarepta Therapeutics’ shares plummeted over 30% on Friday as the company’s approved gene therapy, Elevidys, is under scrutiny by the Food and Drug Administration (FDA). The agency has requested that Sarepta voluntarily stop shipments of the treatment due to concerns over patient deaths.
Elevidys was initially granted conditional approval for patients between 4 and 5 years old with Duchenne muscular dystrophy in 2023, followed by full approval for those 4 and older who can still walk, and accelerated approval for those 4 and up who cannot walk. However, the treatment has yet to clearly prove its benefits for this group.
In a Phase 3 trial, Elevidys failed to meet its goal, although the company argued that it showed promise in other metrics. The FDA’s decision was influenced by head of the Center for Biologics Evaluation and Research, Peter Marks, who agreed with Sarepta’s assessment and overruled FDA staff.
Recent reports have emerged of two teenage boys dying from liver failure after receiving Elevidys, while another person died during a Phase 1 trial of another gene therapy. These deaths raise concerns about the safety risks of Elevidys, particularly given its uncertain benefits. In contrast, Novartis’ gene therapy Zolgensma for spinal muscular atrophy has been shown to have clear benefits despite liver toxicity and death.
Sarepta’s executives have attempted to reassure investors that even if Elevidys can only treat patients who can still walk, the treatment should generate significant revenue, estimated at $500 million per year. However, the company’s stock has fallen over 87% this year due to concerns about the FDA’s potential decision to pull the drug from the market.
One patient, Jennifer Handt, whose son was treated with Elevidys and experienced improvements in his condition, urged caution, stating that families dealing with Duchenne muscular dystrophy would do anything for a treatment option. Despite the risks, she believes every family should have the choice to take Elevidys and potentially see benefits.
Source: https://www.cnbc.com/2025/07/18/sarepta-stock-falls-on-fda-gene-therapy-elevidys-comment.html