PTC Therapeutics has completed a Phase 3 clinical trial of ataluren in patients with nonsense mutation cystic fibrosis (nmCF). The trial is referred to as the Ataluren Confirmatory Trial in Cystic Fibrosis, or ACT CF.

ACT CF was an international, randomized, double-blind, placebo-controlled, efficacy and safety study of ataluren in patients with nmCF not receiving chronic inhaled aminoglycosides. The primary endpoint was lung function as measured by relative change in % predicted FEV1. The study enrolled 208 patients. Each participant was randomly assigned to receive either ataluren three times per day (10-, 10-, 20-mg/kg) or placebo (a substance that looks and tastes like ataluren but does not actually contain the drug), for 48 weeks. It is anticipated that participants who have successfully completed this study will have the opportunity to receive ataluren in an extension study, except in countries where ataluren may be commercially available for the treatment of nmCF.

Additional details about this ataluren clinical trial can be found at or by contacting PTC Therapeutics Patient and Professional Advocacy at (866) 282-5873 or (908) 912-9256 or ACT CF data was announced in March 2017 and can be found in the following press release.

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