Canada Approves Kalydeco for Infants and Children With R117H Mutation

Somi Igbene, PhD avatar

by Somi Igbene, PhD |

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Health Canada has approved Vertex PharmaceuticalsKalydeco (ivacaftor) to treat cystic fibrosis (CF) in patients 4 months to 18 years old who carry the R117H mutation in the CFTR gene, and who weigh at least five kilograms (about 11 pounds).

“It’s been our goal to ensure that as many people with CF as possible are eligible for our treatments, and today’s announcement means that approximately 25 young people with CF in Canada are now newly eligible for KALYDECO,” Michael Siauw, general manager at Vertex Canada said in a press release.

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CF is caused by mutations in the CFTR gene, which produces ‘gate’ CFTR proteins that regulate the flow of water and salts in and out of cells. A CFTR protein that does not work properly causes the build-up of sticky mucus in various organs, such as the lungs, pancreas, intestine, and liver. In patients with a gating mutation, the faulty CFTR gate is stuck closed.

“KALYDECO was first approved in Canada in 2012 as the first medicine to treat the underlying cause of cystic fibrosis in patients with specific mutations,” Siauw said.

Known as a CFTR potentiator, Kalydeco works by keeping the CFTR gate open for longer in patients with a gating mutation. By doing this, Kalydeco is designed to enhance the transport of salt and water across the cell membrane, keeping the airways hydrated and less likely to accumulate sticky mucus.

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The therapy has already been approved in Canada to treat CF patients 18 years and older with the R117H mutation, and those 4 months and older who weigh at least five kilograms and carry one of the following CFTR gene mutations: GG551D, G1244E, G1349, D178R, G551S, S1251N, S1255P, S549N or S549R.

Kalydeco was the first medication to be approved for children as young as 4 months in the U.S. In Europe, it is approved for children 6 months and older who weigh at least five kilograms and carry the R117H mutation or one of the other nine gating mutations mentioned above.

Vertex plans to make the medicine available to newly eligible children in all Canadian provinces and territories as quickly as possible.