FDA Probes Death of 8-Year-Old Linked to Sarepta’s Elevidys Gene Therapy

The U.S. Food and Drug Administration (FDA) is investigating the death of an eight-year-old boy who received Sarepta Therapeutics’ (NASDAQ: SRPT) gene therapy, Elevidys, for Duchenne muscular dystrophy (DMD). The incident occurred on June 7 in Brazil and has triggered renewed scrutiny of the treatment.

Shares of Sarepta fell more than 3% in after-hours trading following the announcement. According to Roche, which holds commercial rights to Elevidys outside the U.S., the boy was not part of a clinical trial. The attending physician assessed the death as unrelated to the therapy, though local health authorities were notified as required. Roche stated it is gathering additional information regarding the case.

Elevidys, a breakthrough therapy for DMD, has faced increasing regulatory challenges. Earlier this year, two non-ambulatory teenage boys died from acute liver failure linked to the therapy. Additionally, a 51-year-old patient treated with Sarepta’s experimental gene therapy SRP-9004 died from the same complication.

In response to the latest fatality, Sarepta announced it will pause Elevidys shipments in the U.S. at the FDA’s request, with Roche halting some international shipments as well. The pause raises questions about the future of the therapy, once hailed as a significant advancement in treating Duchenne muscular dystrophy.

Duchenne muscular dystrophy is a rare, progressive genetic disorder primarily affecting boys, leading to severe muscle weakness and reduced life expectancy. Gene therapies like Elevidys aim to address the underlying genetic cause, offering hope for improved outcomes despite ongoing safety concerns.

The FDA continues to review the incident while Sarepta and Roche cooperate with health authorities. Investors and patients alike await further updates on the therapy’s safety profile and regulatory path forward.