Today an advisory committee of the U.S. Food and Drug Administration (FDA) voted 12 to 1 to recommend that the FDA approve the potential ivacaftor/lumacaftor combination drug (Orkambi).
In some cases, the FDA convenes an advisory committee — a panel of medical experts and community representatives — to provide an independent evaluation of the safety and effectiveness of a potential therapy undergoing review for approval.
While the committee’s recommendation is an important part of the FDA’s review process, its recommendation is not binding. The FDA will make the final decision and has set July 5, 2015, as the target review date.
Orkambi targets the underlying cause of the disease in people with two copies of the F508del mutation. Currently, the FDA is considering whether to approve Orkambi for people with CF ages 12 and older who have two copies of F508del, representing 8,500 people or nearly a third of the CF population in the United States.
Altogether, nearly half of the U.S. CF population has two copies of the F508del mutation. Before the drug combination can be considered as a treatment for children younger than 12, it must be studied in clinical trials for this age group.
In late-stage clinical trials, Orkambi was shown to improve lung function in people with CF and significantly reduce the rate of pulmonary exacerbations, which can lead to frequent hospitalizations and worsening lung function. The drug was developed by Vertex Pharmaceuticals Inc. with significant financial and clinical support from the Foundation.
CF researchers and clinicians testified at the hearing, as did people with CF who participated in clinical trials, sharing firsthand accounts of how the drug has made a difference in their day-to-day lives.
“We are encouraged by the advisory committee’s recommendation today to approve Orkambi for those with two copies of the most common CF mutation,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “We are particularly grateful to the people with CF who participated in the clinical trials as well as to all those from our community who shared powerful stories during today’s hearing.”
“I have waited my entire life for a medical breakthrough that would change what it means to live with this disease,” said Samantha Pelican Monson, a 33-year-old with CF who has two copies of the F508del mutation. “This medication has changed the trajectory of my disease. I no longer live in fear of the future.”
The Foundation’s approach to drug development has led to tremendous advances in the quality of life and life expectancy for people with CF, which has doubled in the past 30 years. Nearly every CF drug available today was made possible because of CF Foundation support.
