Sarepta Halts DMD Shipments After Trial Deaths

Sarepta Therapeutics halted shipments of its Elevidys gene therapy and paused trials for non-ambulatory Duchenne muscular dystrophy patients after two teens died from acute liver failure, prompting the FDA to address the issue with its 'highest level of concern.' The company is developing an enhanced immunosuppressive regimen involving sirolimus to mitigate liver toxicity and is consulting with experts and regulators. Major investment banks, including Goldman Sachs and Morgan Stanley, have downgraded Sarepta and lowered price targets, while Sarepta has withdrawn its 2025 revenue guidance and begun a cost review. Despite the setback, some analysts remain bullish due to the company's liquidity and long-term outlook. Elevidys remains available for ambulatory patients and is the only FDA-approved gene therapy for DMD. The incident has raised industry-wide safety concerns and scrutiny of the FDA's accelerated approval process for gene therapies.