Vertex’s Orkambi (lumacaftor/ivacaftor) Receives Health Canada’s Market Authorization for Children with Cystic Fibrosis Aged 1 to <2 Years
- The company received marketing authorization from Health Canada for expanded use of Orkambi to treat cystic fibrosis in children aged 1-<2yrs. who have 2 copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene
- The approved label expansion was based on the results from the 24wk. P-III study evaluating Orkambi in 46 children aged 1 to <2yrs. which showed that the therapy was found to be well tolerated & the safety profile and PK were similar to prior reported studies in patients aged ≥2yrs.
- The company will collaborate closely with payers to secure access for this new patient population shortly. Orkambi was approved by Health Canada for patients aged ≥2yrs. with CF with 2 copies of the F508del mutation
Ref: Newswire | Image: Vertex
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