Sarepta and Roche have put a trio of Elevidys trials on hold in the EU and UK, pending a complete analysis of a Duchenne muscular dystrophy patient death reported last month.
In a March 31 “community letter” to the World Duchenne Organization, Roche revealed that the European Medicines Agency (EMA) had requested a temporary clinical hold on the studies until “analysis into the cause of death is complete.”
In mid-March, Sarepta had reported that following treatment with Elevidys, a young male patient suffered acute liver failure. While acute liver injury is a known possible side effect of Elevidys — as well as of other AAV-mediated gene therapies — acute liver failure leading to death represents a severity of acute liver injury not previously reported for Elevidys.
According to Sarepta, testing revealed that the patient had a recent cytomegalovirus infection, a common herpes virus infection, which could have been a contributing factor. The infection can damage the liver, resulting in CMV hepatitis.
At the request of the EMA, Roche is now pausing enrollment and dosing of participants in the phase 1 Study 104, phase 2 ENVOL study and the phase 3 ENVISION study at EU country study sites, as well as the UK.
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