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Vertex: KALYDECO Gets European CHMP Positive Opinion For CF In Children

Vertex Pharmaceuticals Inc. (VRTX) announced Friday that KALYDECO (ivacaftor) has received Positive Opinion from European Medicines Agency's or EMA Committee for Medicinal Products for Human Use or CHMP.

The positive opinion is for the label extension of KALYDECO to include the treatment of children and adolescents with cystic fibrosis or CF, ages 6 months
and older weighing at least 5 kg who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator or CFTR gene.

CF is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract.

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 designed to keep CFTR proteins at the cell surface open longer to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.

The European Commission will now review the CHMP's positive opinion. If they issue a favorable adoption, KALYDECO (ivacaftor) will be the first and only approved medicine in Europe to treat the underlying cause of CF in patients ages 6 months and older with the R117H mutation.

In countries where long-term reimbursement agreements have been secured, KALYDECO (ivacaftor), if approved, would be available to eligible patients shortly after Marketing Authorization.

In Germany, the medicine would be available at Marketing Authorization. In all other countries, the company said it will work closely with relevant authorities in Europe to secure access for eligible patients quickly.

In Europe, KALYDECO (ivacaftor) is already approved for the treatment of people with CF ages 18 and older with the R117H mutation, and children ages 6
months and older weighing at least 5 kg who have certain specific mutations in the CFTR gene.

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