Kalydeco: new treatment option for cystic fibrosis

Kalydeco (ivacaftor) is licensed for the treatment of cystic fibrosis in patients aged six years and over with a G551D mutation in the CFTR gene.

The recommended dose of Kalydeco is 150mg every 12 hours with fat-containing food.
The recommended dose of Kalydeco is 150mg every 12 hours with fat-containing food.

PHARMACOLOGY

Ivacaftor is a selective potentiator of the CFTR protein. It increases CFTR channel gating to enhance chloride transport.1 

CLINICAL STUDIES

In two randomised, double-blind, phase III trials in 213 patients with the G551D mutation on at least one allele of the CFTR gene and forced expiratory volume in one second (FEV1) ≥40%, ivacaftor led to a rapid (15 days) improvement in FEV1. The treatment difference between ivacaftor and placebo for mean absolute change in per cent predicted FEV1 from baseline to week 24 was 10.6 percentage points in patients aged 12 years or older and 12.5 percentage points in patients aged 6 to 11 years.1

References:

  1.  Kalydeco Summary of Product Characteristics, August 2012.

View Kalydeco record

Further information: Vertex Pharmaceuticals

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