25 September 2015 - Vertex Pharmaceuticals Incorporated today announced that the European Union Committee for Medicinal Products for Human Use (CHMP) issued a positive Opinion recommending Marketing Authorization of Orkambi (lumacaftor with ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF) in people ages 12 and older with two copies of the F508del mutation. In Europe, approximately 12,000 people with CF ages 12 and older have two copies of this mutation.

The CHMP also issued a positive opinion recommending the indication of Kalydeco (ivacaftor) be expanded to include children ages 2 to 5 with CF, in addition to the current approved use in people ages 6 and older, who have one of nine gating mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N and S549R). A new weight-based oral granule formulation of ivacaftor (50 mg and 75 mg) that is mixed in soft foods or liquids was created for these younger children. The CHMP Opinion for the use of ivacaftor in children ages 2 to 5 included a recommendation to extend weight-based dosing of ivacaftor to children ages 6 to 11 who weigh less than 25 kg using the new oral granule formulation. In addition, the CHMP issued a positive Opinion recommending ivacaftor for use in people with CF ages 18 and older who have the R117H mutation. In Europe, approximately 125 children ages 2 to 5 have one of the nine gating mutations included in today's positive Opinion, and approximately 250 adults have the R117H mutation.

"Today's recommendations bring us closer to being able to help many more people with cystic fibrosis who currently do not have a medicine to treat the underlying cause of this chronic progressive disease," said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex. "However, we know that there are many more people with CF who are still waiting for treatment, and we remain committed to continuing to work toward our goal of creating new medicines for the vast majority of people with cystic fibrosis."

These positive CHMP Opinions are recommendations for approval to the European Commission, which then has the authority to approve medicines for the European Union. The European Commission generally follows the recommendations of the CHMP and typically issues Marketing Authorization within three months. If the CHMP's recommendations are approved, Vertex would then begin the country-by-country reimbursement approval process for each new medicine and indication.

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