Ataluren Proven Effective for Cystic Fibrosis

The trial enrolled 19 patients and showed ataluren induced improvements in chloride channel activity, cystic fibrosis-related cough and positive trends in lung function.

PTC Therapeutics has reported positive clinical results from a Phase II study of ataluren which is used to treat adults with nonsense mutation cystic fibrosis.

The trial, an extension of a short-term open-label Phase IIa proof-of-concept study, enrolled 19 patients and showed that ataluren induced improvements in chloride channel activity, cystic fibrosis-related cough and positive trends in lung function.

The drug also demonstrated combined mean improvements of forced expiratory volume in one second and forced vital capacity.

PTC Therapeutics chief medical officer Robert Spiegel said a 48-week, placebo-controlled Phase III study of ataluren in nonsense