US-based gene therapy company Sarepta Therapeutics will pause shipments of its Elevidys treatment for rare muscle disorders after two teenage boys died from liver failure following the treatment. The US FDA had asked the company to halt the shipments earlier this week, amid safety concerns over a third patient’s death who received an experimental therapy. Sarepta has decided to continue treating ambulatory patients but will temporarily stop all shipments of Elevidys for non-ambulatory patients, citing a need to respond to requests and complete labeling updates. The company’s shares plummeted 8.6% after the announcement.
Source: https://www.cnbc.com/2025/07/21/sarepta-to-pause-gene-therapy-elevidys-shipments-in-us.html