Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, announced an update to the prescribing information for ELEVIDYS , the only approved gene therapy for Duchenne muscular dystrophy (DMD).

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As previously disclosed, the ELEVIDYS label now includes several key updates, including:

  • A boxed warning for the risk of acute serious liver injury (ALI) and acute liver failure (ALF).
  • The non-ambulatory indication has been removed from the Indication and Usage section of the Prescribing Information. The Company expects to quickly commence a study of an enhanced sirolimus immunosuppressive regimen to address the risk of ALI and ALF so that, with the concurrence of the FDA, dosing may resume for non-ambulatory patients.
  • Expanded guidance for prescribers, including a modified pre- and post-infusion oral corticosteroids regimen, and enhanced monitoring recommendations on a weekly basis for 3 months post-infusion.
  • A new Warnings & Precaution regarding increased susceptibility to serious infections due to immunosuppression.

“We want to thank the FDA for their thorough and collaborative review. 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, Ph.D., president of research & development and technical operations.

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To date, ELEVIDYS has been administered to over 1,100 patients globally in clinical and real-world settings. Sarepta continues to work closely with the FDA to ensure that all regulatory decisions are grounded in science and the best interests of patients who face a rare, irreversibly progressive and ultimately fatal disease.

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Source- businesswire