Cystic Fibrosis Drug Could Work In Higher Percentage Of Patients

Vertex Pharmaceuticals’ cystic fibrosis drug, Kalydeco, has had success in mid-stage trials — so much so, in fact, that there is speculation that the number of patients being successfully treated with the drug could double within a few years. Kalydeco is already approved for patients with nine specific genetic mutations. However, the mid-stage trial showed that the drug could be effective in those afflicted with cystic fibrosis due to a residual function mutation, for which the drug had not yet been approved as a treatment. The drug has been successful in the market thus far, with Vertex expecting to collect between $470 and $500 million this year in sales.

Cystic fibrosis is a genetic disorder affecting the lungs. The condition is considered to be dangerous and in many cases life threatening. Vertex has stated that based on the results of the mid-stage trial, the company would begin talks with the FDA and other regulatory bodies regarding a late-stage trial for cystic fibrosis patients with residual function mutations. If the late-stage trial is deemed successful, the drug could be approved.

The drug has also been in the news recently because of Canada’s recent decision to include the drug in the province’s health insurance plan. In Canada,  the sales of the drug could possibly increase after the Ontario Premier Kathleen Wynee said that her province’s health insurance plan will begin covering the costs of the drug. It is estimated that the full year’s course of treatment with Kalydeco would cost upwards of $350,000 a year. About 118 people in Canada are currently taking Kalydeco for the condition. According to The Canadian Press, the Ontario province and Vertex Pharmaceuticals negotiated a deal determining how much the government will pay per dosage. No terms of the agreement were disclosed to the public, but the CBC News says that the deal took months to complete.