The ruling stated that the FDA did not support the approval of the company's New Drug Application (NDA) for aztreonam lysine for inhalation at this time, and suggested that another clinical trial would be necessary for approval.

In response, Gilead issued a press release stating that the company “will continue its dialogue with the FDA to determine whether further analyses of existing data could lead to approval, or whether the company will need to conduct the additional study as stated in the letter.” The discussions are currently underway.

What this means for the CF Community

The CF community is understandably disappointed with this decision. Over 400 CF patients have participated in clinical trials for aztreonam lysine for inhalation since 2003. In addition, more than 250 patients have been receiving the drug through Gilead's Early Access Program (EAP), which is being conducted in nearly 40 CF care centers. 

Because the FDA did not raise any safety issues related to the drug in their ruling, Gilead will continue to provide early access to aztreonam lysine for inhalation for those CF patients with limited treatment options and a serious risk of disease progression. Individuals with CF should contact their care centers to determine their eligibility to participate in the EAP.

Next Steps

The Foundation has met with representatives at Gilead to express its willingness to work with both the company and the FDA to see that aztreonam lysine for inhalation is approved. The Foundation will provide Gilead and the FDA data on the need for new antibiotics for CF and any other relevant information.

The Foundation understands the disappointment of the CF community over the status of Gilead's NDA for aztreonam lysine for inhalation. We will continue to work with all parties involved to seek a timely resolution of this important issue.