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New cystic fibrosis drug shows great promise

VITO PILIECI
Ottawa Citizen
Published on: July 21, 2014

A drug for people with a specific type of cystic fibrosis, whose costs Ontario recently began covering, holds great promise for others with the disease because it’s the first treatment to address the causes rather than just the symptoms, according to supporters.

Kalydeco has been effective in treating patients who suffer from the G551D mutation of the disease, which is a rarer strain of cystic fibrosis. In fact, it’s been touted as the closest thing to a cure. There are about 100 people with this strain in the province of Ontario.

The provincial government started covering the cost of Kalydeco under the Ontario Health Plan in mid-June. While the annual cost of Kalydeco in Ontario was not revealed, the drug can cost up to $300,000 annually for patients in the United States. The drug was developed by Vertex Pharmaceuticals, which is headquartered in Boston.

Kalydeco is among the first cystic fibrosis treatments to be released after years of genetic testing to find out about different mutations of the disease and how each one affects people. By researching each strain, researchers have been able to devise more targeted treatments.

Recent clinical trials have also suggested that, when mixed with other drugs, Kalydeco could provide a long-term treatment for many more cystic fibrosis sufferers.

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