Vertex Pharmaceuticals Inc. (VRTX) said the European Commission has approved KALYDECOTM for people with cystic fibrosis ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator or CFTR gene. KALYDECO is the first medicine to target the underlying cause of the disease in these patients.
Cystic fibrosis is a rare genetic disease caused by defective or missing CFTR proteins resulting from mutations in the CFTR gene.
KALYDECO helps the defective CFTR protein function more normally in patients with the G551D mutation. An estimated 1,100 people in Europe have the G551D mutation. The European Approval is based on positive findings from two global Phase 3 trials in which KALYDECO showed significant and sustained improvements in breathing, weight gain and other measures of disease for people ages 6 and older with this specific genetic mutation, compared to placebo.
Further, people who took KALYDECO were 55 percent less likely to have pulmonary exacerbations, or periods of worsening in the signs and symptoms of the disease that often require treatment with antibiotics and hospital visits, than those who received placebo, the company noted.
The KALYDECO approval comes two months after the European Committee for Medicinal Products for Human Use issued a positive opinion and is the first European approval for Vertex.
by RTT Staff Writer
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