Vertex receives Health Canada approval for cystic fibrosis treatment

Vertex Pharmaceuticals Incorporated has announced that Health Canada has approved KALYDECOTM to treat the underlying cause of cystic fibrosis, or CF, for people ages six and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator, or CFTR, gene.

Cystic fibrosis is a rare genetic disease for which there is reportedly 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.

According to the company, the approval of KALYDECO was based on data from two global phase III 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 less likely to experience pulmonary exacerbations, which are periods of worsening respiratory signs and symptoms that often require treatment with antibiotics and hospital visits, the company said.

The most common serious adverse events included abdominal pain, increased liver enzymes and low blood sugar, which occurred in less than 1 percent of patients. Adverse events commonly observed in those taking KALYDECO included headache, upper respiratory tract infection (common cold), stomach pain and diarrhea. Fewer people in the KALYDECO treatment groups discontinued treatment due to adverse events than in the placebo group. The majority of adverse events associated with KALYDECO were mild to moderate, the company added.

"KALYDECO is an important step toward our ultimate goal of developing new medicines that target the underlying cause of cystic fibrosis for more people with this life-shortening disease," said Peter Mueller, Ph.D., Chief Scientific Officer and Executive vice president of Global R&D at Vertex.

"We are working closely with federal, provincial and territorial governments and private health insurers to bring KALYDECO to all eligible Canadians with cystic fibrosis who have the G551D mutation."

"KALYDECO is a fundamental shift in the way cystic fibrosis is treated because it addresses the underlying cause of the disease, not just its symptoms," said Felix Ratjen, M.D., Division Chief, Respiratory Medicine, The Hospital for Sick Children, and KALYDECO investigator. "In clinical trials, KALYDECO helped people with the G551D mutation breathe more easily and gain weight."