ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy, has received a safety update from Sarepta Therapeutics, the leader in precision genetic medicine for rare diseases. The company also discussed the actions it is taking to improve the safety profile in non-ambulatory patients. Following a second documented instance of acute liver failure (ALF) that resulted in death, these actions were taken. Up till now, both ALF incidents have been non-ambulatory Duchenne patients. Sarepta sends its warmest condolences to the impacted families and caregivers.
At the same time, Roche also revealed today revised dose guidelines for ELEVIDYS (delandistrogene moxeparvovec) that will take effect immediately for patients with non-ambulatory Duchenne muscular dystrophy (DMD), regardless of age, in both clinical and commercial contexts. Elevidys should no longer be administered to non-ambulatory patients in a commercial setting. Enrollment and dosing of non-ambulatory patients in the clinical trial setting will be immediately stopped unless the research protocol incorporates additional risk mitigation strategies (such as immune modulatory medication). Information is shared with doctors, investigators, and health authorities so that patient care can be promptly modified.
Sarepta reports second case of liver failure death after its gene therapy treatment
This decision comes after two cases of fatal acute liver failure (ALF) in non-ambulatory patients were carefully evaluated. Elevidys and other AAV-mediated gene therapies were found to carry a risk, which caused the benefit-risk profile to be reevaluated as unfavorable for non-ambulatory DMD patients. – Read More: Sarepta, Reports 16 Years Old Boy Death After Elevidys Gene Therapy Treatment –
The new dosing restrictions do not impact the treatment of ambulatory DMD patients of any age, and the benefit-risk ratio remains positive in the ambulatory patient population.
The two fatal ALF cases occurred in non-ambulatory patients, out of a total of approximately 140 non-ambulatory patients treated with Elevidys globally to date. Following the first case of fatal ALF, European regulators requested that Roche and Sarepta put temporary clinical holds on Elevidys studies 104 (NCT06241950), 302 (ENVOL, NCT06128564) and 303 (ENVISION Study 303, NCT05310071). The temporary clinical holds are still in effect. Outside of Europe, dosing will be paused, effective immediately, for the ENVISION trial. The dosing restrictions will also go into effect for future dosing of commercial non-ambulatory patients.
Learn More: Frequently Asked Questions About Elevidys
