Overview

• Both fatal cases occurred in non-ambulatory Duchenne muscular dystrophy patients who developed acute liver failure following Elevidys infusion.
• Sarepta has suspended commercial shipments for non-ambulatory individuals and paused dosing in its ENVISION trial for older ambulatory and non-ambulatory patients.
• The company’s stock plunged nearly 40% in early trading after disclosing the second death and its safety response measures.
• The FDA is conducting a high-level review of Elevidys’ safety profile and may require further regulatory actions.
• Elevidys, a $3.2 million one-time gene therapy used by over 900 patients, received accelerated approval despite earlier efficacy concerns.