Vertex’s Orkambi (lumacaftor/ivacaftor) Receives EMA’s CHMP Positive Opinion for Children With Cystic Fibrosis Ages 1 to <2 Years
- The EMA’s CHMP has adopted a positive opinion for the label extension of Orkambi (lumacaftor/ivacaftor) to treat children with CF aged 1-<2yrs. who have two copies of the F508del mutation in the CFTR gene
- The expanded approval was based on the P-III clinical trial evaluating Orkambi in children aged 1-<2yrs. which was found to be safe and well tolerated, and reduced the sweat chloride concentration
- Orkambi was approved in the EU for the treatment of patients with CF aged ≥2yrs. who have two copies of the F508del mutations. If Orkambi is approved, a treatment option will be available for the first time for about 300 young children with CF
Ref: Businesswire | Image: Vertex
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