Vertex Receives EU Approval for ORKAMBI® (lumacaftor/ivacaftor) in Children with Cystic Fibrosis Ages 6-11 with Two Copies of the F508del Mutation

- In Europe, there are approximately 3,400 children ages 6-11 who have two copies of the F508del mutation -

- Existing reimbursement agreements in countries like Ireland will enable rapid access to ORKAMBI; country-by-country reimbursement processes will now begin in other countries-

LONDON – January 10, 2018 –Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted extension of the Marketing Authorization for ORKAMBI® (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF) in people with two copies of the F508del mutation, to include children ages 6 through 11. In Europe, there are approximately 3,400 children ages 6 through 11 with two copies of this mutation. Existing reimbursement agreements in countries such as Ireland will enable rapid access to ORKAMBI for these children. In other countries across the European Union, Vertex will now begin the country-by-country reimbursement process.

“The innovative long-term agreements we have reached in countries like Ireland will enable eligible children to have rapid access to ORKAMBI,” said Simon Bedson, Senior Vice President and International General Manager at Vertex. “Where these agreements are not in place, Vertex is committed to working with local authorities so those who could benefit from this medicine are able to do so as quickly as possible.”

The European Commission’s decision is based on data from two Phase 3 studies of ORKAMBI in children with CF ages 6 through 11 who have two copies of the F508del mutation. In 2017, The Lancet Respiratory published 24-week data from one of these studies, which demonstrated statistically significant improvements in lung function (as assessed by the absolute change in lung clearance index, or LCI2.5, and predicted forced expiratory volume in one second, or ppFEV1) among children treated with ORKAMBI compared to placebo. Improvements in body mass index (BMI) and the Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score were also observed, although they were not statistically significant. Safety data were similar to those observed in an earlier Phase 3 open-label safety study in children ages 6 through 11. The most common adverse events that occurred more frequently among those receiving ORKAMBI compared to placebo were infective pulmonary exacerbation, productive cough, nasal congestion, oropharyngeal pain, abdominal pain upper, headache, upper respiratory tract infection and sputum increased.

“A principal goal of treating CF is slowing the progressive lung damage caused by this life-shortening genetic disease while improving health in the short term,” said Professor Marcus Mall, M.D., Director of the Division of Pediatric Pulmonology & Allergology and the Cystic Fibrosis Center at the Heidelberg University Hospital, Ge