FDA Approves KALYDECO (ivacaftor) as First and Only CFTR Modulator to Treat Eligible Infants With CF as Early as Four Months of Age

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved KALYDECO (ivacaftor) for use in children with cystic fibrosis (CF) ages four months to less than six months old who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to KALYDECO based on clinical and/or in vitro assay data. KALYDECO is already approved in the U.S. and EU for the treatment of CF in patients ages six months and older.

“Since the initial approval of KALYDECO more than eight years ago, we have continued to advance our clinical development program with the goal of treating the underlying cause of cystic fibrosis as early in life as possible,” said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. “Today’s approval is a testament to our relentless efforts, alongside the clinical and scientific community, to reach all people with CF who may benefit from our medicines.”

This FDA approval is based on data from a cohort in the 24-week Phase 3 open-label safety cohort (ARRIVAL) 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). This cohort demonstrated a safety profile similar to that observed in older children and adults.

“Initiating therapy that treats the underlying cause of cystic fibrosis as early as four months of age may have the potential to modify the course of the disease,” said Margaret Rosenfeld, M.D., MPH, Seattle Children’s Research Institute and Department of Pediatrics, University of Washington School of Medicine.

KALYDECO was first approved in 2012 in the U.S. and is now available in more than 40 countries. For more information on KALYDECO, prescribing information or patient assistance programs, visit Kalydeco.com or VertexGPS.com.

About Cystic Fibrosis

Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.