FDA Expands Approval of Vertex’s Orkambi to Include Younger Age Group

The FDA has expanded its approval of Vertex Pharmaceuticals’ Orkambi (lumacaftor/ivacaftor) to include children with cystic fibrosis (CF) between the ages of 12 and 24 months who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

The drug was previously approved for use in people age two years and older who have two copies of the F508del mutation, said the company.

The combined actions of lumacaftor and ivacaftor help hydrate and clear mucus from the airways, the company said.

Lumacaftor is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein. Ivacaftor, which is known as a CFTR potentiator, is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane, said the company.

The agency’s expanded approval was based on results from a phase 3 study in 46 children. Orkambi was generally well-tolerated and the pharmacokinetics and safety profile were similar to those observed in studies in patients age two years and older, according to Vertex. Results were published in the American Journal of Respiratory and Critical Care Medicine.