Ataluren (Translarna™) is a novel, small molecule compound designed to enable production of a full-length and functional CFTR protein in individuals with CF who have nonsense mutations. The compound aims to help the body override a premature signal to stop production of the CFTR protein. 

Status

A phase 3 study completed in 2011 showed that study participants who received ataluren had a lower decline in lung function and a lower rate of pulmonary exacerbations, compared with those who took a placebo. A phase 3 study completed in 2016 did not meet its primary and secondary endpoints of change in lung function and rate of pulmonary exacerbations.

No further clinical development in CF is planned at this time.

Sponsor

This program was sponsored by PTC Therapeutics and partially funded by the Cystic Fibrosis Foundation. The program was conducted within the Therapeutics Development Network.