EU Regulator Rejects Gene Therapy for Rare Disease

Sarepta’s Shares Plummet After European Regulators Reject Elevidys, a gene therapy meant to treat patients with Duchenne muscular dystrophy. The treatment was applied to younger children who could still walk, but regulators cited lack of proof its effectiveness in the disease.

Source: https://www.bloomberg.com/news/articles/2025-07-25/sarepta-roche-gene-therapy-fails-to-get-eu-regulator-approval