Cystic Fibrosis Foundation Celebrates FDA Approval of Orkambi as an Important Advance for the Cystic Fibrosis Community
03 July 2015 - 04:11AM
Business Wire
Drug Targets the Underlying Cause of the
Disease in People with the Most Common CF Mutation
The Cystic Fibrosis Foundation celebrates the FDA’s approval
today of the lumacaftor/ivacaftor combination drug (Orkambi™) 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. For more information, go to
www.cff.org.
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Cystic Fibrosis FoundationLaurie FinkNational Director of Media
Relations301-841-2602lfink@cff.org