Vertex Pharmaceuticals Inc. said the phase 3 study of Kalydeco in cystic fibrosis met its main goal.
Results from the ARRIVAL trial, which assessed children ages 1 to 2 years who have one of 10 mutations in the cystic fibrosis transmembrane conductance regulator, or CFTR, gene, showed that Kalydeco, or ivacaftor, was generally well-tolerated.
The drug's safety data was consistent with results seen in previous phase 3 studies of the treatment in children ages 2 to 5 years and 6 to 11. No patients discontinued treatment due to side effects, the most common of which were cough, fever, elevated liver enzymes and runny nose.
The study also met its secondary goals, showing substantial improvement in sweat chloride, as well as better pancreatic function. The data suggests the potential to modify the course of cystic fibrosis in children as young as 1 year old.
Vertex intends to submit applications for the therapy in children ages 1 to 2 to the U.S. Food and Drug Administration and the European Medicines Agency in the first quarter of 2018.
Kalydeco is approved by the FDA to treat cystic fibrosis in patients ages 2 and up who have one of 38 ivacaftor-responsive mutations in the CFTR gene.
Cystic fibrosis is a rare, life-shortening genetic disease affecting roughly 75,000 people in North America, Europe and Australia.