The US Food and Drug Administration has given approval to ivacaftor (Kalydeco) for treatment of cystic fibrosis in infants aged four months to less than six months who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene.
The FDA approval was based on the results from 24-week phase 3 open-label safety cohort ARRIVAL trial consisting of 6 children with CF ages four months to less than six months who have one of 10 mutations in the CFTR gene (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H). The cohort demonstrated a safety profile similar to that observed in older children and adults.
Cystic fibrosis is a rare, progressive disease that results in the formation of thick mucous that builds up in the lungs, digestive tract, and other parts of the body.
It leads to severe respiratory and digestive problems as well as other complications such as infections and diabetes. Cystic fibrosis is caused by a defective protein that results from mutations in the CFTR gene. While there are approximately 2,000 known mutations of the CFTR gene, the most common mutation is the F508del mutation.