Ataluren is the only treatment currently in clinical development targeting patients with a nonsense mutation. Dr. Jay Barth, Vice President for Clinical Development at PTC Therapeutics hosted a webinar about the study design and timeline for ataluren development. The webinar focussed on the results from the Phase 2b study and set-up of the Phase 3 and open-label extension clinical trials. The European Medicines Agency validated a Marketing Authorization Application seeking conditional approval for ataluren at the end of last year. This was based on the data showing that 174-patients treated with ataluren (10, 10, 20 mg/kg given daily) walked on average 31.3 meters farther than patients on placebo, as measured by the change in six-minute walk distance from baseline to week 48. The primary objective of the multicenter, randomised, double-blind, placebo-controlled Phase 3 study is to confirm the ability of ataluren to slow disease progression as assessed by ambulatory ability based on the 6-minute walk test. Secondary endpoints related to physical function and quality of life will also be assessed. This global study expects to enrol approximately 220 patients. Open-label safety and tolerability studies are also currently taking place for patients who were in previous ataluren clinical trials in both the United States and Europe.
