Sarepta Reverses Course, Pauses Elevidys Shipments Amid FDA Safety Review

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) announced Monday it will voluntarily and temporarily pause all U.S. shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy, effective July 22.

This reversal follows Sarepta's initial refusal to halt distribution despite a Food and Drug Administration (FDA) request amid safety concerns linked to patient deaths.

The pause allows Sarepta and the FDA to complete a safety labeling update, including a black box warning for acute liver injury, and to respond to FDA inquiries.

CEO Doug Ingram emphasized the decision was difficult but necessary to maintain a positive, collaborative relationship with regulators while prioritizing patient safety. The FDA previously placed clinical holds on Sarepta's related gene therapy trials after three deaths potentially associated with their products.

William Blair writes, “While we view the temporary pause of Elevidys shipments as unfortunate, especially for patients that were depending on receiving it in the near term, we believe the decision is necessary to rebuild the company’s relationship with the regulators and the patient community.

The FDA’s initial request also likely put heightened pressure on physicians to pause treatments.

Analyst Sami Corwin said the odds of Elevidys being formally withdrawn from the market “are very low.”

It’s uncertain how long the pause will last. However, William Blair expects Elevidys shipments to restart in the fourth quarter once the label update is complete. The analysts adjusted the forecast to include this delay. The firm estimates Elevidys will bring in $795.7 million in revenue for all of 2025.

For the second quarter, Sarepta expects:

• Net product revenue of $513 million
• Elevidys revenue of $282 million, and
• RNA-based PMOs net product revenue of $231 million.

Amid all the updates regarding Sarepta’s marketed or pipeline gene therapies, Arrowhead Pharmaceuticals Inc. (NASDAQ:ARWR) stock closed 12.2% lower at $14.71.

For the unversed, in November 2024, Arrowhead announced a global licensing and collaboration agreement with Sarepta Therapeutics.

The partnership grants Sarepta exclusive worldwide rights to Arrowhead’s investigational RNA interference (RNAi) therapies for rare genetic diseases targeting muscle, the central nervous system (CNS), and the lungs.

The agreement focuses on Arrowhead’s Targeted RNAi Molecule platform, which enables precise delivery of therapies designed to reduce the activity of disease-causing genes.

Sarepta will pay Arrowhead $500 million and a $325 million equity investment at a 35% premium.

Additional terms include:

• $250 million to be paid in $50 million installments over five years
• up to $300 million in near-term milestone payments over the next year, and
• potential tiered royalties on commercial sales.

Price Action: SRPT stock is down 4.65% at $12.70 during the premarket session at the last check on Tuesday.

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