Cystic Fibrosis Foundation Issues Statement on The New England Journal of Medicine Study on the Benefits of the Ivacaftor/Lumacaftor Combination Drug
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(Bethesda, Md.) -- (Business Wire) -- The Cystic Fibrosis Foundation issued the following statement in response to study results published online today in The New England Journal of Medicine on the clinical trial data for the ivacaftor/lumacaftor combination drug (Orkambi).

“The publication of the Orkambi clinical trial data in The New England Journal of Medicine is a powerful validation that this drug is an important new CF treatment,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “We are proud that the Foundation has played an important role in the drug's development and are especially grateful to the patients, researchers and clinicians who took part in the clinical trials.”

Orkambi targets the underlying cause of the disease in people with two copies of the F508del mutation, which represents nearly half of the U.S. CF population. In late-stage clinical trials, the drug 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 accelerated lung disease.

The publication of the study comes on the heels of a recommendation last week from an advisory committee of the U.S. Food and Drug Administration (FDA) that the FDA approve the drug for people with CF ages 12 and older with two copies of F508del.

Orkambi was developed by Vertex Pharmaceuticals Inc. with significant financial and clinical support from the Foundation.

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.

View The New England Journal of Medicine study and editorial.

About the Cystic Fibrosis Foundation 

The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The CF Foundation funds more cystic fibrosis research than any other organization, and nearly every CF drug available today was made possible because of CF Foundation support. Based in Bethesda, Md., the CF 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: 301-841-2602; lfink@cff.org

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About the CF Foundation | CFTR Modulators | Genetic Therapies
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