Frequently Asked Questions
What is an expanded access program?
The purpose of an expanded access program (EAP) is to make an investigational drug available to those patients with limited or no treatment options before the FDA approval process is complete. To be considered for an EAP, the investigational drug must treat health problems that are serious or life-threatening. To learn more about FDA considerations for an EAP, click here.
How is an expanded access program different than a Phase III clinical study?
The purpose of a Phase III clinical study is to confirm the investigational drug's efficacy, monitor side effects and collect information that will allow the drug to be used safely. The primary goal of an expanded access program is to provide access to the investigational drug for patients with a life-threatening or serious disease, for which there is no good alternative treatment. An expanded access program generally occurs at the end of the clinical development phase, when there is evidence the drug may benefit these patients without exposing them to unreasonable risks. For more information about clinical trials and expanded access programs (or protocols), see clinicaltrials.gov.
What is aztreonam lysine for inhalation?
Aztreonam lysine for inhalation is an antibiotic candidate currently being studied in Phase III clinical trials as a treatment for cystic fibrosis (CF) patients who have pulmonary Pseudomonas aeruginosa. As an investigational drug, aztreonam lysine for inhalation has not yet been determined safe or efficacious in humans by the FDA. Aztreonam lysine has been specifically formulated for inhalation by CF patients.
How is aztreonam lysine for inhalation different than Azactam® (aztreonam for injection)?
Aztreonam for injection is formulated with arginine. Arginine is a substrate for nitric oxide synthetase. Nitric oxide reacts with the superoxide anion to form peroxynitrite, which is pro-inflammatory.1 Aztreonam lysine for inhalation does not contain arginine, but rather incorporates a lysine salt. Additionally, the pH and osmolality of aztreonam lysine are adjusted for inhalation.
What type of data will investigators be required to collect?
Serious adverse event (SAE) data will be collected, but efficacy will not be examined in this EAP. Patients will be evaluated at Screening, Baseline, Day 28, Day 56 and every two months thereafter until discontinuation of the therapy. For an overview of EAP procedures, click here.
Will this EAP be opened to additional patient populations in the future?
Gilead plans to broaden entry criteria in progressive stages. For additional information, visit clinicaltrials.gov and enter "NCT00499720" in the search tool.