Vertex Pharmaceuticals Inc. (VRTX: Quote) announced that the European Committee for Medicinal Products for Human Use, or CHMP, has issued a positive opinion by consensus recommending the approval of KALYDECO (ivacaftor) 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 treat the underlying cause of cystic fibrosis, a rare, genetic disease caused by defective or missing CFTR proteins resulting from mutations in the CFTR gene. In people with the G551D mutation, KALYDECO helps the defective CFTR protein function more normally.
According to Vertex Pharma, the CHMP opinion was based on positive findings from two global Phase 3 studies in which KALYDECO demonstrated unprecedented improvements in breathing and other measures of disease for people ages 6 and older with this specific genetic mutation. People treated with KALYDECO experienced significant and sustained improvements in lung function, weight gain and certain quality of life measurements compared to those on placebo.
Besides, 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 that the CHMP's positive opinion will now be reviewed by the European Commission, which has the authority to approve medicines for the European Union. The European Commission generally follows the recommendation of the CHMP and typically issues marketing approval within three to four months.
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by RTT Staff Writer
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