Kalydeco now approved in Canada for babies 2 months and older

Expanded label covers patients with at least 1 of 10 CF-causing mutations

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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Health Canada has extended its approval of Kalydeco (ivacaftor) to treat children as young as 2 months and weighing at least three kilograms, or about 6.6 pounds, who have cystic fibrosis (CF) caused by mutations that are responsive to the medicine.

The extension covers patients who carry at least one of the following 10 mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or R117H.

Kalydeco was previously approved by Health Canada for use in CF patients, 4 months and older, carrying at least one of nine of those mutations. The R117H mutation was later added to the therapy’s indication in that same age group.

The treatment’s developer, Vertex Pharmaceuticals, will be working with both government and private payers in Canada to help eligible patients get access to the oral medicine as soon as possible.

“For the first time, children with specific mutations will be eligible at 2 months of age for a medicine that treats the underlying cause of their cystic fibrosis,” Michael Siauw, general manager at Vertex Pharmaceuticals (Canada), said in a company press release. “We remain committed to researching and developing medicines for younger CF patients, and this important approval highlights our continued dedication and progress.”

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Health Canada approval follows similar decision in US

The recent label expansion follows a similar decision in the U.S., where Kalydeco is now available for children as young as 1 month old who carry any of the CF-causing mutations from a list of 97.

CF is caused by mutations in the gene that codes for CFTR, a protein that acts like a gate on the surface of mucus-producing cells, helping to control the flow of water and chloride ions in and out of cells. As a result of the mutations, cells produce a thick and sticky mucus that accumulates in several organs, leading to symptoms of CF.

A so-called CFTR potentiator, Kalydeco works by keeping the CFTR gate open for longer. This is expected to help normalize water and chloride ion flow, thereby increasing the water content of mucus, which can be cleared more easily, ultimately easing symptoms.

It was the first approved medicine to address the underlying cause of CF, rather than managing the disease’s symptoms. Kalydeco is available in more than 40 countries around the world, although the eligible ages and mutations may vary from country to country.