Translarna (ataluren) filed in EU for nonsense mutation cystic fibrosis- PTC Therapeutics

The European Medicines Agency (EMA) has validated the submission of a variation for a new indication for Translarna (ataluren), from PTC Therapeutics, for the treatment of nonsense mutation cystic fibrosis (nmCF) for patients not taking chronic inhaled aminoglycoside antibiotics. If approved, ataluren would be the first oral protein restoration treatment that targets the underlying cause of nmCF. Approximately 10% of cystic fibrosis patients have their disease as a result of a nonsense mutation, which can cause the most severe form of cystic fibrosis.

The regulatory application for ataluren for nmCF is based on previously announced analyses from the company's prior completed Phase III double-blind, placebo-controlled study comparing ataluren to placebo in nmCF patients. PTC is conducting an additional randomised, double-blind, placebo-controlled Phase III study of ataluren in nmCF patients and expects enrolment to be completed by the end of 2015, with top-line data expected by the end of 2016.