Vertex Receives CHMP Positive Opinion for KALYDECO for the Treatment of Infants With Cystic Fibrosis Ages 1 Month and Older

Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label expansion of KALYDECO (ivacaftor) for the treatment of infants with cystic fibrosis (CF) ages 1 month to less than 4 months old who have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: R117H, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

“Cystic fibrosis is a chronic, progressive disease that is present from birth, which is why it is important to treat the underlying cause of this disease as early as possible to potentially slow disease progression,” said Fosca De Iorio, Vice President, International Medical Affairs at Vertex. “If the label expansion for KALYDECO is approved, this would allow eligible infants with CF to receive a treatment that targets the underlying cause of their disease at the very start of their lives.”

In the European Union, KALYDECO is already approved for the treatment of people with CF ages 4 months old and above with specific mutations in the CFTR gene.

About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 92,000 people globally. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands 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, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein 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, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the 30s, but with treatment, projected survival is improving.