Vertex' Kalydeco marks a milestone in fight against cystic fibrosis

The oral drug ivacaftor (VX-770) provides major, sustained improvement in lung function, growth and other signs and symptoms for cystic fibrosis (CF) patients, according to results of the pivotal Phase III clinical trial published in the New England Journal of Medicine. The multi-site study marks the first time a drug has been shown to work on the disease process rather than on the symptoms. CF, which has no cure, is the most common lethal genetic disease in Caucasians.

Developer of the drug, USA-based Vertex Pharmaceuticals (Nasdaq: VRTX), recently filed a New Drug Application with the Food and Drug Administration for Kalydeco (ivacaftor), a medicine in development that targets the defective protein that causes cystic fibrosis (The Pharma Letter October 20).

"Ivacaftor represents a significant advancement in the treatment of CF. This study shows that the therapy can safely provide long-term benefits to patients with a specific type of cystic fibrosis," said Michael Konstan, one of the study's co-authors and Chairman of the Department of Pediatrics at Case Western Reserve University School of Medicine and UH Rainbow Babies and Children's Hospital, noting: "The availability of this medication for these CF patients holds great promise."