Ontario, Canada (CU)_ Kalydeco (ivacaftor) has been licensed by Health Canada for the treatment of cystic fibrosis (CF) in individuals who have the R117H mutation in the CFTR gene and who are aged four months to eighteen years and weigh a minimum of five kilos (about 11 pounds).
Michael Siauw, general manager at Vertex Canada released a media statement saying, “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”. According to Siauw, “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”.
The major cause of cystic fibrosis is mutations in the CFTR gene, which generates the gate CFTR proteins that control the passage of water and salts in and out of cells. A dysfunctional CFTR protein results in the accumulation of sticky mucus in several organs, including the lungs, pancreas, gut, and liver. The defective CFTR gate remains closed in patients with a gating mutation.
Kalydeco, also referred to as a CFTR potentiator, functions by keeping the CFTR gate open for an extended period of time in patients with a gating mutation. Through this process, Kalydeco facilitates the passage of salt and water across the cell membrane, thereby maintaining the airways hydrated and less prone to mucus accumulation.
The medicine is already licensed in Canada for the treatment of patients with cystic fibrosis who are 18 years and above and having the R117H mutation, and kids who are aged four months and above and weighs at least five kilos and have one of the following CFTR gene mutations: GG551D, G1244E, G1349, D178R, G551S, S1251N, S1255P, S549N, or S549R.
Kalydeco was the first drug to be licensed in the United States for children aged as young as four months. It is authorized in Europe for children aged six months and above who weigh a minimum of five kilos and have the R117H mutation or one of the other nine gating mutations described above. Vertex intends to make the medication immediately accessible to newly eligible youngsters across all Canadian provinces and territories at the earliest possible.
