Vertex Pharmaceuticals Receives Health Canada’s Approval for KALYDECO™

Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) announced that KALYDECO™, which it describes as the first medicine to treat the underlying cause of cystic fibrosis, has been approved by Health Canada for people aged six and older who have a specific type of gene mutation.

As quoted in the press release:

Cystic fibrosis is a rare genetic disease for which there is no cure. It is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. In people with the G551D mutation, KALYDECO (kuh-LYE-deh-koh) helps the defective or missing CFTR protein function more normally. Approximately 100 people in Canada with CF are believed to have this mutation.

The approval of KALYDECO was based on data from two global Phase 3 studies of people with CF who have at least one copy of the G551D mutation. Those who were treated with KALYDECO experienced significant and sustained improvements in lung function and weight gain compared to those who received placebo. In one study, people who took KALYDECO were also significantly less likely to experience pulmonary exacerbations, which are periods of worsening respiratory signs and symptoms that often require treatment with antibiotics and hospital visits.

Click here to read the full Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) press release.