Sarepta Therapeutics announced an update to the prescribing information for its Duchenne muscular dystrophy gene therapy, Elevidys.
The label now includes a boxed warning for the risk of acute serious liver injury (ALI) and acute liver failure (ALF) — a detail previously shared by Sarepta in July, following the deaths of two non-ambulatory patients.
The non-ambulatory indication has been removed from the indication and usage section of the therapy’s prescribing information. Sarepta says it will quickly launch 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.
Additional updates include:
- Expanded guidance for prescribers, including a modified pre- and post-infusion oral corticosteroids regimen, and enhanced monitoring recommendations on a weekly basis for three months post-infusion.
- A new ‘warnings & precaution’ regarding increased susceptibility to serious infections due to immunosuppression.
Sarepta says it will continue 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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