FDA issues approval for Gileads's Cayston for respiratory symptoms in cystic fibrosis

Gilead Sciences, Inc. (Nasdaq:GILD) today announced that the U.S. Food and Drug Administration (FDA) has granted marketing approval for Cayston^® (aztreonam for inhalation solution) as a treatment to improve respiratory symptoms in cystic fibrosis (CF) patients with Pseudomonas aeruginosa (P. aeruginosa). Cayston’s safety and efficacy have not been established in pediatric patients below the age of 7, patients with forced expiratory volume in one second (FEV₁) of less than 25 percent or greater than 75 percent predicted, or patients colonized with Burkholderia cepacia.

“We look forward to making Cayston available to the cystic fibrosis community as soon as possible.”

Cayston is administered at a dose of 75 mg three times daily over a 28-day period and is delivered via the Altera^® Nebulizer System, a portable, drug-specific delivery device using the eFlow^® Technology Platform, developed by PARI Pharma GmbH. PARI Pharma also contributed to the development of Cayston’s drug formulation for delivery with the Altera Nebulizer System. Cayston will be available in the United States by the end of next week through certain specialty pharmacies.

“All of us at Gilead extend our thanks to the investigators and to the people with cystic fibrosis who took part in the Cayston clinical trials,” said Norbert Bischofberger, PhD, Gilead’s Executive Vice President, Research and Development and Chief Scientific Officer. “We look forward to making Cayston available to the cystic fibrosis community as soon as possible.”

CF is a chronic, debilitating genetic condition that affects the respiratory and digestive systems of approximately 70,000 people worldwide, including 30,000 people in the United States. Chronic respiratory tract infection with P. aeruginosa contributes to the decline in pulmonary function, which is often associated with morbidity and mortality among CF patients.

“Since its founding in the 1950s, the Cystic Fibrosis Foundation has worked to advance the care and treatment of cystic fibrosis and we are pleased with the progress to date,” said Robert J. Beall, PhD, President and Chief Executive Officer, Cystic Fibrosis Foundation. “However, a significant need for new treatments remains for people with cystic fibrosis, particularly for those with chronic pseudomonal infection. As the first new inhaled antibiotic approved for use in cystic fibrosis in more than a decade, Cayston therefore represents an important therapeutic option in the care of patients with cystic fibrosis.”

Cayston received conditional marketing authorizations in the European Union and Canada in September 2009 and was approved in Australia in January 2010. Applications for marketing approval of Cayston are currently pending in Switzerland and Turkey.

Reimbursement and Access to Care

Gilead also announced today the establishment of a program designed to minimize barriers to access for Cayston for uninsured, privately insured and government-insured people with cystic fibrosis.

Additionally, Gilead is launching the Cayston^® Access Program, a call center developed with Cystic Fibrosis Foundation Pharmacy, LLC, a wholly owned subsidiary of the Cystic Fibrosis Foundation. The program will assist people with cystic fibrosis and members of their care team with insurance verification, referral to participating specialty pharmacies, claims support and co-pay assistance. For information about the Cayston Access Program, call 1-877-7CAYSTON (877-722-9786) or visit www.cayston.com.

Important Safety Information

To reduce the development of drug-resistant bacteria and maintain the effectiveness of Cayston and other antibacterial drugs, Cayston should only be used to treat patients with CF known to have P. aeruginosa in the lungs.

Cayston is contraindicated in patients with a known allergy to aztreonam.

Severe allergic reactions have been reported following administration of aztreonam for injection to patients with no known history of exposure to aztreonam. In addition, allergic reaction with facial rash, facial swelling and throat tightness was reported with Cayston in clinical trials. If allergic reaction to Cayston does occur, stop administration of Cayston and initiate treatment as appropriate.

Caution is advised when administering Cayston to patients if they have a history of beta-lactam allergy, although patients with known beta-lactam allergy have received Cayston in clinical trials and no severe allergic reactions were reported. A history of allergy to beta-lactam antibiotics such as penicillins, cephalosporins, and/or carbapenems may be a risk factor, since cross-reactivity may occur.

Bronchospasm is a complication associated with nebulized therapy, including Cayston. Reduction of 15 percent or more of FEV₁ immediately following administration of study medication after pretreatment with a bronchodilator was observed in 3 percent of patients treated with Cayston.

In clinical trials, patients with increases in FEV₁ during a 28-day course of Cayston were sometimes treated for pulmonary exacerbations when FEV₁ declined after the treatment period. Healthcare providers should consider a patient’s baseline FEV₁ measured prior to Cayston therapy and the presence of other symptoms when evaluating whether post-treatment changes in FEV₁ are caused by a pulmonary exacerbation.

Prescribing Cayston in the absence of known P. aeruginosa infection in patients with CF is unlikely to provide benefit and increases the risk of development of drug-resistant bacteria.

Adverse reactions occurring in more than 5 percent of patients treated with Cayston compared to placebo in Phase III studies were cough (54 percent versus 51 percent), nasal congestion (16 percent versus 12 percent), wheezing (16 percent versus 10 percent), pharyngolaryngeal pain (12 percent versus 11 percent), pyrexia (13 percent versus 6 percent), chest discomfort (8 percent versus 6 percent), abdominal pain (7 percent versus 5 percent) and vomiting (6 percent versus 4 percent).