Duchenne Muscular Dystrophy gene therapy distribution suspended
Sarepta has voluntarily paused all shipments of its Duchenne muscular dystrophy gene therapy Elevidys in the U.S.
The FDA made the distribution suspension request Friday after two patient deaths following treatment with Elevidys. More recently, Sarepta disclosed the death of a third patient who was given one of its gene therapies. That patient had received an investigational therapy for a different disease—limb-girdle muscular dystrophy (LGMD)—in a clinical trial.
This pause in shipments will allow Sarepta the necessary time to respond to any requests for information and allow Sarepta and FDA to complete the ELEVIDYS safety labelling supplement process. The company is working with the FDA to include a black box warning for acute liver injury and an updated risk management scheme in the drug’s label.
“It is important for the patients we serve that Sarepta maintains a productive and positive working relationship with FDA, and it became obvious that maintaining that productive working relationship required this temporary suspension while we address any questions that FDA may have and complete the Elevidys label supplement process,” said Sarepta CEO Doug Ingram.
