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Vertex : CHMP Grants Positive Opinion For KAFTRIO - KALYDECO In People Ages 12 And Older With CF

Vertex Pharmaceuticals Inc. (VRTX) said that the?European Medicines Agency's or EMA Committee for Medicinal Products for Human Use or CHMP adopted a positive opinion for KAFTRIO in a combination regimen with KALYDECO or ivacaftor 150 mg to treat people with cystic fibrosis or CF ages 12 and older with one F508del mutation and one minimal function mutation or two F508del mutations in the cystic fibrosis transmembrane conductance regulator or CFTR gene.

If granted Marketing Authorization, the company said that the people ages 12 and older in Europe who have one F508del mutation and one minimal function mutation will for the first time be able to benefit from a medicine that treats the underlying cause of the disease.

The company noted that people 12 years of age and older who have two F508del mutations also will be eligible for the new triple combination regimen.

Cystic Fibrosis is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF.

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