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Children with CF in Canada as young as age 1 OK’d for Orkambi

About 30 children expected to gain access to treatment: Vertex

Health Canada has extended its approval of Orkambi (lumacaftor/ivacaftor) to treat children as young as 1 who have cystic fibrosis (CF) caused by two copies of the F508del mutation.

With this approval, about 30 children in Canada are expected to gain access to treatment that addresses the root cause of their disease, according to Orkambi’s developer Vertex Pharmaceuticals. The company said it will be working with payers in Canada to get eligible patients access as soon as possible.

“With this approval, we are delighted that this new patient group is eligible for a medicine that treats the underlying cause of their disease. This approval enables us to advance closer toward our goal of developing medicines for people of all ages living with CF,” Michael Siauw, general manager, of Vertex in Canada, said in a company press release.

CF is caused by mutations in the gene that provides instructions for making the CFTR protein. Everyone inherits two copies of the gene, one from each parent. The most common CF-causing mutation is F508del and just under half the patients in the Canadian Cystic Fibrosis Registry carry two copies of the gene carrying this specific mutation.

The CFTR protein normally acts like a gate on the surface of the cell, helping to control the flow of chloride ions into and out of it. The F508del mutation causes the protein to become unstable so it can’t get to the cell’s surface.

Orkambi contains a combination of two molecules called CFTR correctors that help restore the protein’s function — one molecule helps stabilize the CFTR protein so it can get to the surface, the other helps keep the gate-like protein open once it’s at the cell surface. Data from clinical trials have indicated it can help slow lung function decline and improve nutritional outcomes for people with CF caused by two F508del mutations.

Orkambi was previously available in Canada for CF patients ages 2 and older. The U.S. Food and Drug Administration approved a similar expansion for patients as young as 1 last year.

Expanded approval for these young patients was supported by data from a Phase 3 clinical trial (NCT03601637) that enrolled 46 infants and toddlers, ages 1 to just under 2. Participants were treated with Orkambi for about six months and results suggested its safety and pharmacological profiles were similar in them as in older patients. Orkambi also reduced chloride levels in patients’ sweat, indicating improved CFTR protein function, data showed.

“As a trial investigator, I have seen the benefit Orkambi can bring to children ages 1-2 living with cystic fibrosis,” said Larry Lands, MD, PhD, an investigator on the trial at McGill University. “This approval is a great step towards initiating eligible patients earlier to potentially slow the progression of the disease.”



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