The Food and Drug Administration granted conditional approval Thursday to the first gene therapy for Duchenne muscular dystrophy. Regulators restricted the treatment to younger patients, with additional data required to broaden its use.
The gene therapy, called Elevydis, is made by Sarepta Therapeutics. The company did not immediately release a price but the one-time treatment is expected to be among the most expensive medicines ever.
“The approval of Elevidys is a watershed moment for the treatment of Duchenne. Elevydis is the first and only gene therapy approved for Duchenne, and this approval brings us closer to our goal of bringing forward a treatment that provides the potential to alter the trajectory of this degenerative disease,” said Doug Ingram, Sarepta’s president and chief executive officer.