PTC sales and marketing update

The U.K.’s NICE issued final guidance recommending the use of Translarna ataluren from PTC to treat nonsense mutation Duchenne muscular dystrophy (DMD) in ambulatory patients aged five years and older - its approved indication. In April, the agency issued draft guidance and recommended Translarna contingent upon a discount under a managed access agreement. The recommended dose is 40 mg/kg per day, with an annual cost of L220,256 ($291,200) for a 24-26 kg patient (see BioCentury, April 25). ...