Kalydeco Approved in Canada to Treat Infants 4 Months and Older

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Health Canada has extended the use of Kalydeco (ivacaftor) to treat babies, starting at 4 months old, who have cystic fibrosis (CF) caused by certain mutations.

The specific gating mutations covered are: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. Kalydeco, Vertex Pharmaceuticals, was previously approved in Canada to treat children, starting at age 1, and adults with these mutations.

“With today’s approval, children in Canada as young as 4 months now have a medicine to treat the underlying cause of their disease,” Nia Tatsis, executive vice president and chief regulatory and quality officer at Vertex, said in a press release.

“This is another step in our goal to develop medicines to treat people living with CF as early in life as possible,” Tatsis added.

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Vertex will work with payers to ensure that infants who are now eligible for Kalydeco are able to access the therapy, the company stated. Each Canadian province and territory is responsible for bringing treatments into its health insurance plan, and prescription medications can be acquired by residents through that plan or through private insurance.

CF is caused by mutations in the gene CFTR, which provides instructions for making a protein of the same name. Normally, the CFTR protein acts like a “gate” in the cell’s membrane, helping to regulate the flow of water and salts in and out of cells, which is important for the formation of mucus.

Different types of mutations in the CFTR gene lead to different effects on the CFTR protein; one type of mutation is called a gating mutation. As the name suggests, this kind of mutation causes the “gate” in the CFTR protein to be “stuck closed.”

Kalydeco is a CFTR potentiator, a type of CF treatment that works to open the gate in the CFTR protein, thereby normalizing the transport of salts and water in and out of cells.

Health Canada’s expansion of Kalydeco’s approval follows similar decisions in the U.S. and in Europe.

These approvals were supported by data from an ongoing Phase 3 clinical trial called ARRIVAL (NCT02725567). This open-label trial (meaning all enrolled are given Kalydeco), sponsored by Vertex, includes six children with eligible mutations between the ages of 4 and 6 months. It is due to conclude in July 2022.

The medication’s safety profile in these infants has been consistent with that reported in older age groups.