Vertex ($VRTX) has taken another step toward building a global franchise for treating cystic fibrosis. European regulatory advisers backed approval of the trailblazing drug Kalydeco for a subset of patients with the genetic disorder, paving the way for a likely go-ahead to market the drug in the EU.
Cambridge, MA-based Vertex has been working to expand the commercial reach of the CF drug, which is the first to treat the underlying cause of the disease. The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) endorsed the company's application to market Kalydeco to treat CF patients with the G551D mutation, which is found in about 1,100 patients in Europe. The FDA approved the drug for such patients in January.
Cystic fibrosis causes the lungs to fill with sticky mucus and leads to frequent pulmonary infections and early death. Vertex's drug revamps the function of CFTR protein, and the treatment showed in multiple late-stage studies that it could improve breathing in patients with the G551D mutation, which about 4% of CF patients carry. Seeking to treat a larger percentage of the CF population, Vertex has been testing drug combos such as Kalydeco and the experimental VX-809.
About half of the world's CF patients reside in Europe, where about 35,000 people have the disease. The European Commission will consider the CHMP's recommendation in its final decision on Kalydeco.
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