Positive Phase 3 Study Of ORKAMBI® In Children With Cystic Fibrosis Ages 6-11 Who Have Two Copies Of The F508del Mutation Supports A Submission To The European Medicines Agency In The First Half Of 2017

Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) today announced the results of a Phase 3 study of ORKAMBI ® (lumacaftor/ivacaftor) in children with cystic fibrosis (CF) ages 6 through 11 who have two copies of the F508del mutation. The study met its primary endpoint of absolute change in lung clearance index (LCI 2.5) through 24 weeks of treatment, demonstrating a statistically significant improvement in LCI 2.5 among patients treated with ORKAMBI compared to placebo. LCI is a sensitive measure of lung function in early CF disease and the European Medicines Agency (EMA) agreed to the primary endpoint for this study. In the first half of 2017, Vertex plans to submit a Marketing Authorization Application (MAA) line extension to the EMA for the use of ORKAMBI in this patient population. Data from a previous Phase 3 open-label safety study in children ages 6 through 11 supported the U.S. Food and Drug Administration approval of ORKAMBI in September 2016. In this second study, ORKAMBI was well tolerated with safety data that were similar to data from the previous Phase 3 study. There are approximately 3,400 children ages 6 through 11 who have two copies of the F508del mutation in Europe.

"This study is an important complement to recently presented long-term data in patients 12 years and older suggesting ORKAMBI may modify the course of CF. These new data demonstrate that treating the underlying cause of the disease with ORKAMBI improves lung function in even younger patients," said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex. "We are preparing to submit these important data to the EMA in the first half of 2017, and we look forward to bringing ORKAMBI to eligible children in Europe as soon as possible."

"CF is a progressive disease that begins at birth, and traditional measurements do not always detect the early lung damage that occurs in children," said Felix Ratjen, M.D., Division Chief of Pediatric Respiratory Medicine at The Hospital for Sick Children Toronto, Professor of Pediatrics at The University of Toronto, a Senior Scientist at the Research Institute in the Department of Physiology and Experimental Medicine, and Principal Investigator for the study. "LCI is a sensitive measure of lung function, and these new data demonstrate that treating children early with ORKAMBI can improve lung function."

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