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CAMBRIDGE, Mass. - Sarepta Therapeutics, Inc. (NASDAQ:SRPT) announced Friday that the prescribing information for ELEVIDYS, its gene therapy for Duchenne muscular dystrophy (DMD), has been updated with several key safety modifications. The company's stock, currently trading at $19.35, has experienced significant volatility, having fallen over 83% in the past year according to financial data.
The updated label now includes a boxed warning for the risk of acute serious liver injury and acute liver failure. Additionally, the non-ambulatory indication has been removed from the Indication and Usage section of the prescribing information. This regulatory setback comes as InvestingPro analysis shows the company is quickly burning through cash, with negative free cash flow of $451.54 million in the last twelve months.
The revised guidance for healthcare providers includes a modified pre- and post-infusion oral corticosteroids regimen and enhanced monitoring recommendations on a weekly basis for three months post-infusion. A new warning regarding increased susceptibility to serious infections due to immunosuppression has also been added.
"Completion of the safety labeling change for ELEVIDYS will ensure that families and healthcare professionals have clear information, supported by a Medication Guide, to help understand these updates and guide treatment decisions," said Louise Rodino-Klapac, president of research & development and technical operations at Sarepta.
According to the company, ELEVIDYS has been administered to over 1,100 patients globally in clinical and real-world settings. Sarepta stated it plans to quickly commence a study of an enhanced sirolimus immunosuppressive regimen to address the liver injury risks, with the goal of resuming dosing for non-ambulatory patients with FDA concurrence.
ELEVIDYS is currently indicated for ambulatory patients 4 years of age and older with DMD who have a confirmed mutation in the DMD gene. The single-dose, adeno-associated virus-based gene therapy is designed to address the underlying genetic cause of Duchenne muscular dystrophy.
The information in this article is based on a press release statement from Sarepta Therapeutics.
In other recent news, Sarepta Therapeutics reported its third-quarter earnings for 2025, exceeding expectations with an earnings per share of -$0.13, compared to the forecasted -$0.7. The company also achieved a revenue of $399 million, surpassing the projected $337.91 million by 18.2%. These results highlight the company's strong performance in the quarter. Meanwhile, the U.S. Food and Drug Administration maintained approval for Sarepta's gene therapy, Elevidys, for ambulatory patients four years and older with Duchenne muscular dystrophy, despite adding a Boxed Warning due to safety concerns. In analyst updates, Mizuho upgraded Sarepta Therapeutics from Neutral to Outperform, citing strong demand for Elevidys and favorable insurance coverage. Conversely, Baird lowered its price target for the company to $15.00, following the failure of the ESSENCE trial, described as the longest and largest dystrophin replacement study. These developments reflect the evolving landscape for Sarepta Therapeutics in the pharmaceutical industry.
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