Overview
- The updated U.S. label highlights a risk of potentially fatal liver failure for the Duchenne muscular dystrophy treatment.
- Use is now restricted to walking patients aged four or older with a confirmed DMD gene mutation.
- The FDA advises weekly liver-function testing for the first three months after infusion.
- Regulators acted after two non-ambulatory teens died of acute liver failure, and the agency halted shipments in July during a safety review.
- Sarepta shares rose following the label change, while Europe’s CHMP has recommended against approving the therapy.