Cystic fibrosis drug from Vertex is approved by Canadian regulators

Vertex Pharmaceuticals Inc. said Monday that Canadian regulators have given their approval to Kalydeco, a drug designed to treat some patients suffering from cystic fibrosis, a rare and often fatal genetic disease.

In January, Cambridge-based Vertex received a similar approval for the drug from the Food and Drug Administration in the United States. Kalydeco was OK’ed by European regulators in July.

In Monday’s press release, Vertex said that Health Canada has approved Kalydeco to treat people ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator gene. About 100 people in Canada with cystic fibrosis are believed to have this mutation.

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Other cystic fibrosis drugs on the market only treat symptoms of the inherited disease, which causes mucus to thicken and block the passage of air to the lungs, making it harder for patients to breathe and digest food. Kalydeco targets the cause of the disease in roughly 4 percent of the US patient population of 30,000, a Feb. 1 story in the Globe noted.

Kalydeco was discovered as part of a collaboration with Cystic Fibrosis Foundation Therapeutics Inc., the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation.

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