Drug Development Pipeline
Restore CFTR Function
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.
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.
Recent Ataluren Studies
Completed with Results
Latest News on Ataluren
June 08, 2012
PTC Therapeutics Inc. today announced results from a Phase 3 clinical trial of ataluren, a potential oral drug aimed at treating the underlying cause of cystic fibrosis.
September 10, 2009
PTC Therapeutics announced today the start of a Phase 3 clinical trial of an oral drug targeted at treating the underlying cause of cystic fibrosis.
August 20, 2008
Sign up for clinical trial alerts
Get email updates about clinical trials that matter to you.
Explore clinical trials
Be a part of the movement transforming the future of cystic fibrosis treatment.Learn More