FDA Approves Duchenne Muscular Dystrophy Treatment for Children

FDA has approved an orphan drug for treatment of the most common form of muscular dystrophy in children.

The agency approved Emflaza, a corticosteroid tablet used to treat Duchenne muscular dystrophy in patients 5 and older. Corticosteroids are a common treatment for the disorder, but Emflaza, marketed by Illinois-based Marathon Pharmaceuticals, is the first such product approved for use in young children.

The FDA fast-tracked the application and prioritized its review as an orphan drug. The agency issued the sponsor a rare pediatric disease review voucher — only the ninth voucher issued to date — as part of a program aimed at speeding the approval for products for treating rare diseases in children. Congress renewed the program in December as part of the 21st Century Cures Act.