Gilead cystic fibrosis treatment reaches trial goal

The drug reached its primary efficacy endpoint of change at day 28 of the trial from baseline in respiratory symptoms, as assessed by the Cystic Fibrosis Questionnaire-Revised (CFQ-R), a patient-reported outcome tool used to measure health-related quality of life for people with the disease.

This is the first phase III clinical trial in cystic fibrosis (CF) to evaluate patient-reported outcomes as a primary endpoint, the US biopharmaceutical company said.

The randomized study involved a 28-day treatment course with 75 mg of aztreonam lysine for inhalation or a volume-matched placebo administered three times daily. Data from the study showed that aztreonam lysine demonstrated a significant improvement in the CFQ-R.

Aztreonam lysine-treated patients also experienced significant improvements at day 28 in respiratory function. The most common treatment-emergent adverse events in the study were cough, productive cough, nasal congestion, sore throat and shortness of breath, but these were not significantly different between the two groups, the company said.

A Bruce Montgomery, senior vice president and head of respiratory therapeutics at Gilead Sciences, said: “Based on the successful completion of our second pivotal study, we plan to submit a new drug application for aztreonam lysine for inhalation for the treatment of people with CF who have pulmonary pseudomonas aeruginosa to the US FDA in the second half of 2007.”

Gilead said that data from the study has not been reviewed by the FDA and added that aztreonam lysine is an investigational compound and has not yet been determined safe or efficacious in humans.