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Vertex Receives Two EU Approvals for KALYDECO® (ivacaftor) for People with Cystic Fibrosis
-Country-by-country reimbursement processes will now begin-
"These approvals bring us closer to our goal of developing new medicines
to treat the underlying cause of cystic fibrosis for as many people as
CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. In people with gating mutations, or an R117H mutation, the CFTR protein reaches the cell surface but does not work properly. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to help CFTR proteins at the cell surface open more often to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.
Ivacaftor in Children Ages 2 to 5 with Gating Mutations
"We know that the progressive damage caused by cystic fibrosis can start
at birth, so early treatment is critical to offering the best chance of
improving long-term outcomes," said Professor
The European Commission's Decision is based on previously announced results of a 24-week open-label Phase 3 study that was designed to evaluate the safety and pharmacokinetics of weight-based dosing of ivacaftor (50 mg or 75 mg twice daily) in children ages 2 to 5. A weight-based oral granule formulation of ivacaftor that is mixed in soft foods or liquids was created for these younger children and is available in two strengths, 50 mg and 75 mg. The approval also includes an extension of weight-based dosing of ivacaftor to children ages 6 to 11 who weigh less than 25 kg, using the new oral granule formulation.
"Expanding the use of ivacaftor will allow younger children with cystic
fibrosis to benefit from earlier treatment of the underlying cause of
their disease," continued
Ivacaftor in Adults with an R117H Mutation
"While people with an R117H mutation can exhibit a wide range of
severity in their CF, once their disease begins to progress, lung
function decline can be severe," continued
The European Commission's Decision is based on previously announced data from a Phase 3 study of ivacaftor that enrolled 69 people with CF who had an R117H mutation.
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 cystic fibrosis transmembrane conductance regulator (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. The median age of death is in the mid-to-late 20s.
Ivacaftor is the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene. Known as a CFTR potentiator, ivacaftor is an oral medicine that aims to help the CFTR protein function more normally once it reaches the cell surface, to help hydrate and clear mucus from the airways.
For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu once posted.
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. Ivacaftor and lumacaftor 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, as amended,
including the quotes in the second, fourth, sixth and seventh paragraphs
of this press release and statements regarding the country-by-country
reimbursement approval process. While the company believes the
forward-looking statements contained in this press release are accurate,
there are a number of factors that could cause actual events or results
to differ materially from those indicated by such forward-looking
statements. Those risks and uncertainties include, among other things,
risks related to commercializing ivacaftor in these patient populations
and the other risks listed under Risk Factors in Vertex's annual report
and quarterly reports filed with the
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