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

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.