Did The FDA Make A Mistake? Sarepta's Elevidys Approval Under Scrutiny

The Food and Drug Administration (FDA) on Tuesday said it is investigating the risk of acute liver failure with serious outcomes, including hospitalization and death, following Sarepta Therapeutics, Inc.’s SRPT Elevidys treatment and evaluating the need for further regulatory action.

Since approval, the agency has received two reports of fatal acute liver failure following treatment of non-ambulatory pediatric male patients with Duchenne Muscular Dystrophy (DMD) with Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus vector-based gene therapy.

The two reported deaths in non-ambulatory DMD patients appear to be related to treatment with Elevidys. Reports were received from clinical trial and postmarketing data.

Also Read: Sarepta Therapeutics Stock Plummets Over 40% On Second Fatal Case Linked To Its Gene Therapy

In these two cases, the patients presented with elevated transaminases and were hospitalized less than two months after treatment with Elevidys.

On Wednesday, HC Wainwright reiterated its Sell rating for Sarepta, maintaining a price forecast of $10. Analyst Mitchell Kapoor stated his belief that “it is highly likely that the FDA will at least require updated language to the USPI to denote the risk of liver failure or death.”

The analyst says the worst-case scenario for Sarepta would be if the FDA ultimately required the company to withdraw Elevidys from the U.S. market.

Kapoor underscored a critical risk for Sarepta, stating, “If Elevidys were to be withdrawn, we see little intrinsic value remaining in the company.”

Kapoor further highlighted the FDA as a potential headwind, noting that Center for Biologics Evaluation and Research (CBER) head Vinay Prasad has previously voiced strong disapproval of Peter Marks’ initial approval of Elevidys.

This established skepticism from Dr. Prasad, even prior to the recent reports of liver failure deaths, suggests a likely reluctance to support Sarepta’s continued marketing of Elevidys.

For a broader perspective on Sarepta’s outlook, explore how other analysts view the stock.

Price Action: SRPT stock is trading lower by 9.96% to $17.09 at last check Wednesday.

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