(Bethesda, Md.) -- The Cystic Fibrosis Foundation celebrates the FDA's approval today of the lumacaftor/ivacaftor combination drug (Orkambi^TM) as an important advance for the cystic fibrosis community. Orkambi targets the underlying cause of the disease in people with two copies of the most common CF gene mutation, F508del.

The FDA approved the drug for people with CF ages 12 and older who have two copies of the F508del mutation, representing 8,500 people or nearly one-third of the CF population in the United States. Nearly half of individuals with CF have two copies of this mutation and could eventually benefit from the therapy.

Orkambi was developed by Vertex Pharmaceuticals Inc. with significant clinical, scientific and funding support from the CF Foundation.  

“We applaud the FDA for its swift approval of Orkambi,” said Robert J. Beall, president and CEO of the CF Foundation. “It is our hope that everyone who is prescribed this drug will have immediate access to it so they can begin to live longer, healthier lives.”

Beall continued: “We are proud of the important role that the Foundation played in the research and development of both ivacaftor and lumacaftor, including initiating the first efforts that led to the discovery and development of these small molecules.”

Orkambi improves lung function and significantly reduces the rate of pulmonary exacerbations, which can lead to frequent hospitalizations and accelerated lung disease. It is the second drug approved by the FDA that targets the underlying cause of CF. Ivacaftor (as a single therapy) was the first.  

Cystic fibrosis is a life-threatening genetic disease that causes serious lung infections and premature death. It affects 30,000 children and adults in the United States and 70,000 people worldwide. People with the disease inherit a defective gene that causes a buildup of thick mucus in the lungs, pancreas and other organs.

In people with two copies of the F508del mutation, a series of problems prevents a protein, called CFTR, from achieving the correct shape and reaching the cell surface where it is needed to help regulate the flow of salt and fluids in and out of the cells of the lungs and other organs.

Orkambi combines lumacaftor and ivacaftor to treat these problems with a two-step approach. Lumacaftor helps move the defective CFTR protein to its proper place at the cell surface. Ivacaftor increases the activity of the protein once it is there, supporting the flow of salt and fluids, which helps thin the thick mucus that builds up in the lungs of people with CF.

“While the approval of Orkambi is a meaningful step forward, we won't lose sight of the promises we've made to all people with cystic fibrosis and their families,” said Preston W. Campbell, III, M.D., executive vice president for medical affairs of the CF Foundation. “We will continue to fight the disease from every angle, exploring all promising avenues of research, including those that are pioneering new technologies to repair the defective CF gene, so that one day all people with CF will no longer have the disease.”

Orkambi is the latest example of the CF Foundation's venture philanthropy model, through which it has invested hundreds of millions of dollars to help develop CF drugs and therapies. This approach has led to tremendous advances in 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.

In addition to supporting research programs with Vertex, the CF Foundation invests in the CF research programs of many leading pharmaceutical companies, including Pfizer, Genzyme and Shire, to accelerate the discovery and development of new drugs that will help more people with cystic fibrosis.

About the Cystic Fibrosis Foundation

The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more cystic fibrosis research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The Cystic Fibrosis Foundation is a donor-supported nonprofit organization. 

Media Contact

Laurie Fink, National Director of Media Relations, Cystic Fibrosis Foundation: 301-841-2602; lfink@cff.org