-CHMP recommends Marketing Authorization for lumacaftor in combination with ivacaftor for people with cystic fibrosis ages 12 and older with two copies of the F508del mutation-
-CHMP recommends Marketing Authorization for ivacaftor for children with cystic fibrosis ages 2 to 5 with 9 gating mutations & for people with cystic fibrosis ages 18 and older with the R117H mutation-
The CHMP also issued a positive Opinion recommending the indication of
KALYDECO® (ivacaftor) be expanded to include children ages 2
to 5 with CF, in addition to the current approved use in people ages 6
and older, who have one of nine gating mutations in the cystic fibrosis
transmembrane conductance regulator (CFTR) gene (G551D,
G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N and S549R).
A new weight-based oral granule formulation of ivacaftor (50 mg and 75
mg) that is mixed in soft foods or liquids was created for these younger
children. The CHMP Opinion for the use of ivacaftor in children ages 2
to 5 included a recommendation to extend weight-based dosing of
ivacaftor to children ages 6 to 11 who weigh less than 25 kg using the
new oral granule formulation. In addition, the CHMP issued a positive
Opinion recommending ivacaftor for use in people with CF ages 18 and
older who have the R117H mutation. In
"Today's recommendations bring us closer to being able to help many more
people with cystic fibrosis who currently do not have a medicine to
treat the underlying cause of this chronic progressive disease," said
These positive CHMP Opinions are recommendations for approval to the
About Cystic Fibrosis
Cystic fibrosis is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.
CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic, or genotyping test, lead to CF by creating non-working or too few CFTR protein at the cell surface. The defective function or absence of CFTR proteins in people with CF results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. Today, the median predicted age of survival for a person with CF is between 34 and 47 years, but the median age of death remains in the mid-20s.
Collaborative History with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT)
Vertex initiated its CF research program in 1998 as part of a collaboration with CFFT, the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation. Lumacaftor and ivacaftor were discovered by Vertex as part of this collaboration.
Vertex is a global biotechnology company that aims to discover, develop and commercialize innovative medicines so people with serious diseases can lead better lives. In addition to our clinical development programs focused on cystic fibrosis, Vertex has more than a dozen ongoing research programs aimed at other serious and life-threatening diseases.
Founded in 1989 in Cambridge, Mass., Vertex today has research and
development sites and commercial offices in the United
States, Europe, Canada and
Special Note Regarding Forward-looking Statements
This press release contains forward-looking statements as defined in the
Private Securities Litigation Reform Act of 1995, including, without
limitation, statements regarding the
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