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.
“This is an indication of things to come. Kalydeco is now being studied in combination with two different products. That will be effective for a majority of the cystic fibrosis population,” said Ken Chan, vice-president of advocacy, research and health care at Cystic Fibrosis Canada.
He said the combination treatment could benefit as many as 2,000 people suffering from cystic fibrosis as early as next year.
“What we are pleased and excited about is that this is the first drug that not only helps to improve the quality of life, but is a drug that targets the underlying causes of cystic fibrosis. Most medicines address the symptoms but not the cause.”
Cystic fibrosis is a genetic disease that affects multiple systems in the body, with the lungs and digestive system being the most severely affected, making eating and breathing difficult. It is the most common fatal genetic disease affecting Canadian children and youth. Half of the people who died of the disease in 2011 were under the age of 34.