The U.S. health regulator has granted accelerated approval to Sarepta Therapeutics’ first-of-its-kind gene therapy for Duchenne muscular dystrophy (DMD), an inherited progressive muscle-wasting disorder that almost always affects young boys.
Sarepta said on Thursday the Food and Drug Administration had approved the treatment for children aged between 4 and 5 years who can walk. It was initially seeking approval for all DMD patients who can walk.
The therapy’s wholesale acquisition cost is $3.2 million, CEO Douglas Ingram said on a conference call with investors.
The world’s most expensive treatment, a gene therapy for bleeding disorder hemophilia from Australian drugmaker CSL Ltd, carries a list price of $3.5 million….