Sarepta Therapeutics Stock Tumbles 42% Following Second Drug‑Linked Death Report

Shares of Sarepta Therapeutics Stock tumbled 42% after the biotech company reported a second death connected to its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys. The news rattled investors, erasing market value and dragging the stock to nine-year lows.

This second fatality has raised serious concerns over the safety of the drug, especially for non-ambulatory patients. Investors are now questioning the future of Elevidys and the company’s regulatory standing, leading to a sharp market sell-off.

Background on Elevidys and Its Approval

Elevidys gained accelerated FDA approval in 2023 as the first gene therapy for DMD. The treatment targets children four years and older, delivering dystrophin-producing genes via a modified virus.

The FDA later expanded its use to non-ambulatory patients despite mixed trial outcomes. Each single infusion carries a $3.2 million price tag.

What Triggered the Plunge?

• Second fatality confirmed: A teenager experienced acute liver failure similar to the one reported in March. Both patients were non‑ambulatory and had higher dosages.
• Market reaction: Following the news, Sarepta shares dropped about 42%, later estimates show a near 45% decline, hitting a nine‑year low around $18.30–$21.
• Trial and shipment halt: Sarepta paused dosing in its Phase III ENVISION trial for non-ambulatory patients and suspended shipments, cooperating with an FDA review.

Market and Analyst Response

Wall Street analysts expressed alarm:

• Regulatory Risk: With two serious adverse events, tighter FDA restrictions, or even withdrawal for some patients, could follow. 
• Downgrades: Several firms cut their ratings. Piper Sandler lowered its recommendation from “Overweight” to “Market Perform,” and H.C. Wainwright shifted to “Sell,” dropping targets substantially.
• Revenue uncertainty: Elevidys was expected to contribute over $2 billion in 2025 revenue. With suspensions and questions around safety, those projections are now in doubt. 

Company Response and Next Steps 

Sarepta has taken swift action:

1. Independent expert panel: The company is convening experts to recommend new immunosuppressive protocols to reduce liver injury risk.
2. Regimen Adjustments: Plans include adding drugs like sirolimus to control the immune response.
3. Ongoing FDA corporation: Any changes must gain FTA approval, a process now under heightened scrutiny.
4. Financial recalibration: Revenue guidance for 2025 has been pulled to reassess projections and spending.

Investor Takeaways

• High volatility expected: After tumbling 42–45%, shares could swing sharply in either direction depending on trial updates, FDA decisions, or expert panel findings.
• Regulatory risk looms: The FDA, under new leadership (e.g., Dr. Vinay Prasad), may take a stricter approach to gene therapies like Elevidys.
• Long-term outlook in flux: If immune suppression measures prove effective, Elevidys could recover. Without them, market confidence and price targets may fall further.
• Gene therapy sentiment at stake: This incident adds pressure across the gene therapy space, as safety concerns may impact investor sentiment in adjacent biotech players.

Conclusion 

The news that Sarepta Therapeutics’ Stock tumbled 42% reflects deep investor concern over patient safety and regulatory response. While the company is trying to stabilise therapy protocols and comply with FDA oversight, Elevidys’s path forward is uncertain. For now, investors should monitor trial developments, safety data, and FDA actions closely.

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This content is made for learning only. It is not meant to give financial advice. Always check the facts yourself. Financial decisions need detailed research.