Vertex Pharmaceuticals’ cystic fibrosis treatment received European approval for use in patients with a rare gene mutation.
The Cambridge, Mass., company said Friday that the European Commission approved the drug for patients ages 6 and older who have at least one copy of the mutation in the cystic fibrosis transmembrane conductance regulator gene.
Cystic fibrosis is a disease that causes sticky mucus to build up in the lungs and other organs, which leads to infections, digestive problems and death in young adulthood. It is caused by defective or missing proteins from mutations in the gene, and according to research by Vertex Pharmaceuticals Inc., its Kalydeco drug helps the defective protein function more normally.
Studies of the pill showed it significantly improved lung function and reduced other symptoms of cystic fibrosis.
The U.S. Food and Drug Administration approved Kalydeco in January as a treatment for patients with that gene mutation.