Translarna ataluren: Completed Phase III enrollment

PTC completed enrollment of about 220 patients ages 7-16 years with nonsense mutation DMD in the double-blind, placebo-controlled, international Phase III ACT DMD trial evaluating oral Translarna thrice daily for 48 weeks. Patients will receive 10 mg/kg Translarna in the morning and midday and 20 mg/kg in the evening. Top-line data are expected in 2H15.

Last month,the European Commission granted conditional approval to Translarna to treat nonsense mutation DMD. As a condition of the approval, PTC will need to complete ACT DMD (see BioCentury, Aug. 11). PTC plans to launch Translarna in selected countries in 1H15. In July, PTC launched an expanded access program for Translarna. PTC said funded named-patient programs have already been authorized in France, Turkey and Spain (see BioCentury, July 21). ...