The European Commission has approved Vertex Pharmaceuticals‘ oral medicine Kalydeco (ivacaftor) for people with cystic fibrosis (CF).
Kalydeco is for CF patients who have one of eight non-G551D gating mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
In July 2012, the medicine first received European approval for CF patients of ages six and older with the G551D mutation, considered to be the most common gating mutation.
The Committee for Medicinal Products for Human Use (CHMP) expressed a positive opinion on the medicine in June 2014.
The eight additional gating mutations in the latest approval are G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D.
Cystic fibrosis is a result of either a defective or missing CFTR protein caused by mutations in the CFTR gene. CFTR proteins serve as channels at the cell surface that control the flow of salt and water into and out of the cell.
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However, in people with CF problem, the CFTR protein at the cell surface becomes defective and causes abnormally thick, sticky mucus in the lungs. The digestive tract and several other organs are also affected.
Kalydeco regulates the CFTR protein to function normally once it reaches the cell surface.
Targeting the abnormal CFTR protein channels, the oral medicine helps open the channels to enable chloride ions to move into and out of the cell, which in turn helps thin the mucus and hydrate and protect the airways.
The latest approval is based on the data collected from the first part of a Phase III, two-part, randomised, double-blind, placebo-controlled, cross-over study of 39 CF patients ages six and older who have a non-G551D gating mutation.
In the first part of the study, patients treated with Kalydeco showed improvements in lung function (FEV1), sweat chloride, body mass index and CFQ-R scores, and in the second part these improvements were maintained throughout all 24 weeks of treatment.
Vertex senior vice president and international general manager Simon Bedson said: "We are committed to working closely with the appropriate national authorities to make Kalydeco available for these patients as soon as possible."
The drug is also approved in Australia, New Zealand and Switzerland for people with CF who have at least one copy of the G551D mutation in the CFTR gene.