FDAnews Drug Daily Bulletin
Pharmaceuticals / Submissions and Approvals

European Commission Approves Cystic Fibrosis Treatment for Children

Jan. 29, 2019
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The European Commission approved a label extension for Vertex Pharmaceuticals’ Orkambi (lumacaftor/ivacaftor) for treatment of children with cystic fibrosis.

The new approval applies to patients aged two to five years old who have two copies of the F508del mutation, the most common form of the rare genetic disease.

The Commission granted approval based on data from a Phase 3 study in 60 patients that tolerated the treatment generally well for 24 weeks. The safety profile in the pediatric patients was “generally consistent” with the safety profile in patients six and older, Vertex said.

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