Health Canada Approves KALYDECO™ (ivacaftor), The First Medicine To Treat The Underlying Cause Of Cystic Fibrosis In People With A Specific Genetic Mutation (G551D)

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.

“Health Canada’s approval of KALYDECO is a welcome first step to getting Canadian CF patients access to this important advance in treatment,” said Ken Chan, Vice President, Advocacy, Research and Healthcare of Cystic Fibrosis Canada. “We are pleased that advances in CF research have led to the development of innovative, personalized new medicines such as KALYDECO. We look forward to working with Vertex and Canada’s publicly-funded drug plans to provide patients with access to KALYDECO.”

The gene that causes CF was identified in 1989 as a result of collaborative research led by Lap-Chee Tsui, Ph.D., and Jack Riordan, Ph.D., at The Hospital for Sick Children in Toronto and Francis Collins, M.D., Ph.D., at the University of Michigan.

KALYDECO was discovered as part of a collaboration with Cystic Fibrosis Foundation Therapeutics, Inc., the non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation.

About the Canadian Funding Process

Canadian approval and reimbursement of a new medicine is a multi-step process. Once a new medicine receives Notice of Compliance (NOC), or approval, from Health Canada, it goes through the Common Drug Review (CDR) process that conducts a cost-benefit analysis. Each province and territory then conducts a review and makes its own reimbursement decision using the CDR recommendation as a guide.

About KALYDECO

KALYDECO™ (ivacaftor) is the first treatment to target the underlying cause of CF in people with the G551D mutation in the CFTR gene. Known as a CFTR potentiator, KALYDECO is an oral medicine that aims to help the CFTR protein function more normally once it reaches the cell surface, to help hydrate and clear mucus from the airways. KALYDECO (150mg, q12h) was first approved by the U.S. Food and Drug Administration in January 2012, by the European Medicines Agency in July 2012 and by Health Canada in November 2012 for use in people with CF ages 6 and older who have at least one copy of the G551D mutation in the CFTR gene.

Vertex retains worldwide rights to develop and commercialize KALYDECO. KALYDECO is under review by the Therapeutic Goods Administration (TGA) of Australia. KALYDECO™ is a trademark of Vertex Pharmaceuticals Incorporated and has been authorized for use by Vertex Pharmaceuticals (Canada) Incorporated in Canada.

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