Drug Approved to Treat Duchenne Muscular Dystrophy

The FDA overruled the recommendations of its own advisory committee to approve Exondys 51 (eteplirsen) to treat Duchenne muscular dystrophy, a rare and lethal form of the disease that affects about 15,000 Americans, reports Kaiser Health News (KHN). The rocky road to approval is something Managed Care has been following for awhile.

KHN: “Exondys 51 doesn’t cure Duchenne muscular dystrophy and will only help a minority of patients. It is designed for the 13 percent of patients with a particular genetic mutation that prevents them from making dystrophin, a key protein that keeps muscles intact.”

Insurers and PBMs note that it’s going to be expensive, at $350,000 a year.