PTC sales and marketing update

The U.K.’s NICE issued draft guidance recommending the use of Translarna ataluren from PTC to treat nonsense mutation Duchenne muscular dystrophy (DMD) in ambulatory boys aged five years and older. The recommendation is contingent on PTC providing Translarna at an undisclosed discount under a managed access agreement. NICE estimated Translarna’s annual cost at L220,256 ($311,134) for a 24-26 kg patient. NICE expects to issue final draft guidance for Translarna in May. ...