Health Canada approval signals shift in CF treatment for newborns 

NOVEMBER 30, 2023

Health Canada has approved the cystic fibrosis drug Kalydeco (Ivacaftor) for children aged 2-4 months old who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N,S549R or R117H.  

Based on birth data from previous years, Cystic Fibrosis Canada estimates that this change will impact approximately five babies each year. While this approval will impact a small number of families, it is a significant development in the treatment of CF.  

Newborn screening for cystic fibrosis is standard practice for all newborns in Canada and with this Health Canada approval, if a child receives a positive diagnosis for cystic fibrosis from a newborn screening, they will be able to start on a treatment almost immediately after diagnosis. 

“This is the start of a paradigm shift in the treatment of cystic fibrosis in Canada,” said Dr. Paul Eckford, Chief Scientific Officer, Cystic Fibrosis Canada. “We hope to see more children in Canada start on a modulator therapy as soon as they are diagnosed through newborn screening, preventing unnecessary decline and enabling these kids to grow up with as little interruption to life from cystic fibrosis as possible.” 

What is Kalydeco? 

Kalydeco is a precision medicine for the treatment of cystic fibrosis. It is one of the drugs that makes up the triple combination drug, Trikafta. Kalydeco is a pill taken twice a day, with fat-containing food, that helps the protein made by the CFTR gene function better and, as a result, improves lung function and other aspects of cystic fibrosis such as increasing weight. 

Who can benefit from Kalydeco? 

In Canada, the number of people with mutations eligible for Kalydeco is over 200, or 4% of the cystic fibrosis population. Kalydeco is approved in Canada, for the treatment of patients with cystic fibrosis (CF) who have one of the following mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R or R117H, and who are:  

• 2-4 months of age and weighing at least 3 kg 4 months and older and weighing 5 kg to less than 25 kg  
• 6 years and older and weighing 25 kg or more