international clinical trials published in
the New England Journal of Medicine, a
combination of two drugs has shown
encouraging results for those who suffer
from cystic fibrosis Caused by genetic
mutations, Cystic fibrosis is an incurable
lung disease that causes the body to
overproduce thick mucus, leading to
chronic lung infections and pancreatic
problems.
Combined lumacaftor-ivacaftor therapy
was shown to be safe and effective for
cystic fibrosis patients with two copies of
the cystic fibrosis gene mutation
(F508del) found in nearly half of the
patients with this disease.
The two drugs are called ivacaftor and
lumacaftor. Ivacaftor is already approved
by the U.S. Food and Drug Administration
for treating certain people with cystic
fibrosis, according to the researchers.
"Just a few years ago, ivacaftor became
the only FDA-approved drug for the
genetic defect in cystic fibrosis, but it only
works for genetic mutations found in a
small portion of cystic fibrosis patients.
Our study showed that combining
ivacaftor with lumacaftor helps patients
with the most common cystic fibrosis
mutation. This is an exciting step
forward," said study co-author Dr.
Susanna McColley in a news release from
the Ann & Robert H. Lurie Children's
Hospital of Chicago.
She is the associate director of the
hospital's Cystic Fibrosis Center. Cystic
fibrosis is a genetic disease that causes
thick, sticky mucus to form in the lungs,
pancreas and other organs.
In 2012, Ivacaftor, known by the brand
name Kalydeco, made by Vertex
Pharmaceuticals, became the first drug to
win FDA approval for CF after studies
indicated that it helped people who have a
more rare genetic mutation for CF that is
found in only about 4% of all CF patients.
Lumacaftor, also made by Vertex
Pharmaceuticals is yet to get approval.
According to Vertex, an FDA advisory
panel voted 12-1 to approve their
combination, branded as Orkambi.
In the two trials, which studied over 1,100
patients with the diseased from six
different countries from April of 2013 to
April of 2014, showed the drug helped to
achieve an improvement of 3% in function
of the lung over the use of a placebo.
The randomized, double-blind, placebo-
controlled clinical trial included a total of
1,108 patients, 12 years of age and older,
who were treated for 24 weeks in multiple
centers.
Orkambi should help more than 14,000
patients with cystic fibrosis in the United
States with the two copies of the mutant
gene.
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