European Commission Approves KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat Children With Cystic Fibrosis Ages 6 to 11 Years

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted approval for the label extension of KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in patients ages 6 through 11 years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

“We are delighted that KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor is now approved for these young patients in the European Union. It provides a new treatment option for physicians to help treat the underlying cause of this devastating disease early in life,” said Reshma Kewalramani, M.D., Chief Executive Officer and President at Vertex. “This important milestone brings us one step closer to our ultimate goal of developing treatments for all patients living with CF.”

“Ivacaftor/tezacaftor/elexacaftor plus ivacaftor has shown clinical benefit since its availability last year for people with CF ages 12 and above,” said Professor Marcus A. Mall, M.D., Head of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine at Charité University Medical Center Berlin. “CF is a progressive disease, in which symptoms and organ damage manifest very early in life. As a physician, I welcome the approval of this medicine for this younger age group, as it will help us treat eligible children with CF as early as 6 years old.”

As a result of long-term reimbursement agreements in Austria, Northern Ireland and Denmark, and provisions for access in health care systems such as Germany, eligible patients in these countries will have access to the expanded indication for KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor shortly following regulatory approval by the European Commission. Vertex will continue to work with reimbursement bodies across the European Union to ensure access for all eligible patients.

This medicine has also been approved by regulatory authorities in New Zealand and in Switzerland, where it is known as TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor), for people with CF ages 6 and above, and we continue to work closely with reimbursement bodies in these countries to ensure access for all eligible patients.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 83,000 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 the 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 early 30s.