Gilead applies to FDA for approval of respiratory treatment for cystic fibrosis patients

FOSTER CITY, Calif. Gilead Sciences announced that it has filed a new drug application to the Food and Drug Administration for marketing approval of an investigational therapy in development for people with cystic fibrosis who have pulmonary infection.

The therapy, known as aztreonam lysine for inhalation, is delivered by the eFlow Electronic Nebulizer. “Chronic pseudomonal airway infection represents the single greatest cause of morbidity and mortality for people with cystic fibrosis, and with a limited number of inhaled antibiotics, there remains a significant unmet medical need,” said A. Bruce Montgomery, senior vice president and head of respiratory therapeutics at Gilead Sciences. “The completion of this new drug application is reflective of Gilead’s commitment to developing novel therapies for people with this life-threatening disease.”

Data from the first clinical trial revealed that patients with cystic fibrosis experienced improvement in respiratory symptoms, according to measurements taken by the Respiratory Symptoms scale of the Cystic Fibrosis Questionnaire-Revised, a patient-reported outcome tool used to measure health-related quality of life for people with cystic fibrosis.

The second clinical trial concluded that the therapy significantly delayed the time to need for inhaled or intravenous antibiotics following a course of inhaled tobramycin. Both studies also demonstrated improvements from baseline in respiratory function, as measured by relative improvement of forced expiratory volume in one second compared to a placebo.

The most common treatment-emergent adverse events in the AIR-CF studies were cough, productive cough, nasal congestion, wheezing and sore throat. The incidences of these events were not significantly different between the placebo and the aztreonam lysine for inhalation groups.

In August 2007, Gilead initiated an expanded access program to provide aztreonam lysine for inhalation to people with cystic fibrosis and P. aeruginosa who have limited treatment options and are at risk for disease progression. Aztreonam lysine for inhalation is an investigational therapy and has not yet been determined safe or efficacious in humans.