Vertex Receives CHMP Positive Opinion for ORKAMBI for Children with Cystic Fibrosis

Vertex announced that the European Union Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending extension of the Marketing Authorization for ORKAMBI (lumacafator/ivacaftor) to children with cystic fibrosis (CF) ages 6 through 11 who have two copies of the F508del mutation. There are approximately 3,400 patients ages 6 through 11 eligible for this medicine in Europe.

"Cystic fibrosis is a systemic, multi-organ, progressive disease present from birth," said David Gillen, M.D., Head of International Medical Affairs at Vertex. "This recommendation brings us closer to being able to help more people with CF who currently do not have a medicine to treat the underlying cause of their disease."

Positive CHMP opinions are recommendations for approval to the European Commission, which has the authority to approve medicines for the European Union. The European Commission generally follows the recommendations of the CHMP and typically extends the Marketing Authorization within three months. In some countries, we have in place existing agreements that will make ORKAMBI available to this age group immediately following Marketing Authorization. In other countries, Vertex will begin the country-by-country reimbursement process following Marketing Authorization.

Cystic fibrosis is a rare, life-shortening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working or too few CFTR protein at the cell surface. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell 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 mid-to-late 20s.