Vertex Pharmaceuticals announced that Health Canada, as a partner of the Access Consortium, has granted Marketing Authorization for the expanded use of ORKAMBI for the treatment of cystic fibrosis in children ages 1 to less than2 years who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene This approved label expansion is based on data from a 24-week, Phase 3, open-label, multi-center study in 46 children ages 1 to less than 2 years who have two copies of the F508del mutation. ORKAMBI was generally well tolerated, and the safety profile and pharmacokinetics were similar to that observed in studies in patients ages 2 years and older. Vertex will work closely with payers to secure access for this new patient population as soon as possible.
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