Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) shares saw a handy gain on Thursday after the U.S. Food and Drug Administration (FDA) gave the nod to Vertex’s cystic fibrosis (CF) treatment. Essentially, the FDA approved Kalydeco (ivacaftor) to include use in children diagnosed with CF ages 12 to 24 months who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene.
This FDA approval is based on data from the ongoing Phase 3 study (ARRIVAL) of 25 children with CF aged 12 to 24 months who have one of 10 mutations in the CFTR gene. The study demonstrated a safety profile consistent with that observed in previous Phase 3 studies of older children and adults; most adverse events were mild or moderate in severity, and no patient discontinued due to adverse events.
Kalydeco already was approved in the United States for the treatment of CF in patients ages 2 and older who have one of 38 ivacaftor-responsive mutations in the CFTR gene.
Looking ahead, Vertex submitted a Marketing Authorization Application for a line extension (ages 12 to 24 months) to the European Medicines Agency, with a decision anticipated in the first half of 2019.