Sarepta's Gene Therapy Death Prompts Guidance Suspension and Stock Plunge

Patient Death and Gene Therapy Concerns

• 💔 A 15-year-old patient died from liver failure after receiving Sarepta's gene therapy, Elevus, for Duchenne muscular dystrophy.
• ⚠️ This is the second death in recent months, with a 16-year-old boy also dying after receiving the same treatment.
• 🧬 Both patients who died had progressed to a stage where they were no longer able to walk, a key distinction from the initial FDA approval.

FDA Approval and Controversial Expansion

• 📜 The FDA initially approved Elevus for younger patients (4-5 year olds) who could still walk.
• 📈 The agency controversially expanded the label last year to include nearly all Duchenne patients, despite less evidence for efficacy in older, more advanced cases.

Sarepta's Response and Financial Impact

• 🚚 Sarepta has halted shipments of the therapy for more advanced patients while investigating a new safety regimen.
• 📉 CEO Doug Ingram announced the suspension of Sarepta's 2025 full-year guidance, with an updated outlook to be provided later.
• 📊 Analysts had projected $1.5 billion in Elevus sales for the year, but these projections are now in serious doubt.
• 📉 The company's stock has plummeted, down approximately 48% today and 85% over the past year, reflecting growing concerns.