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FDA expands approval of first gene therapy for rare form of muscular dystrophy

·1 min

The US Food and Drug Administration has approved the use of the first gene therapy to treat a rare form of muscular dystrophy in most people who have the disease and a specific genetic mutation. The drug, called Elevidys, was initially approved for children ages 4 and 5 with Duchenne muscular dystrophy and a confirmed mutation in the DMD gene. The FDA has now given traditional approval for ambulatory people 4 and older with the DMD gene mutation, and accelerated approval for non-ambulatory people in the same age group. The therapy costs about $3.2 million per patient. Duchenne muscular dystrophy causes progressive muscle weakness primarily in boys and currently has no cure. Elevidys provides another treatment option and is administered as a one-time intravenous infusion.