$300,000 a year drug approved in Canada
BY Staff | December 4, 2012
For CF patients with a specific G551D mutation, the drug helps to improve the function of the defective protein, leading to better lung function, weight gain and lower sweat chloride levels. The G551D mutation is present in roughly 100 Canadians with cystic fibrosis.
One of World's Most Expensive Drugs
Kalydeco is one of the most expensive drugs in the world. Treatment currently costs US$300,000 a year. Representatives of the drug's manufacturers, Vertex Pharmaceuticals Inc., says the Canadian price will be approximately the same.
Cystic Fibrosis Canada has publicly called on Vertex Pharmaceuticals Inc. and drug plan administrators to work together to resolve the high price of Kalydeco so that CF patients can access this drug in an equitable and affordable way.
"The approval of Kalydeco is just the first step in bringing this new, breakthrough therapy to CF patients," said Ken Chan, vice-president, advocacy, research and healthcare for Cystic Fibrosis Canada. "As one of the most expensive drugs in the world, CF patients simply cannot afford to pay for this much-needed medicine. Cystic Fibrosis Canada is calling for a compassionate approach from manufacturers and drug plan administrators when it comes to making new, life-changing medicines accessible to CF patients."
According to a recent article, Cystic Fibrosis Canada is supporting the Vertex's application to the Common Drug Review, hoping that body will recommend that provinces add Kalydeco to their drug formularies.
Doctors call for access
Cystic Fibrosis Canada isn't the only one asking for help getting this drug into patients hands.
"Kalydeco is an incredible leap forward in CF therapy," said Dr. Larry Lands, CF clinic director at Montreal Children's Hospital. "Even though Kalydeco as a stand-alone treatment will only help a limited segment of affected patients, it shows promise as a combination therapy for other patients with cystic fibrosis, and points the way towards the development of tailored treatments that will correct the underlying problem for each and every affected person with cystic fibrosis."
"This treatment represents a paradigm shift in therapy; it's the first one to target the basic defect in the cells of CF patients," said Dr. Elizabeth Tullis, head of respirology and director of the adult cystic fibrosis program at St. Michael's Hospital in Toronto. "While Kalydeco is currently applicable only to a small proportion of CF patients; it gives us hope that a similar approach can be developed for all people with cystic fibrosis."
"The future of some babies diagnosed through CF newborn screening is going to be revolutionized by Kalydeco," said Dr. Mark Chilvers, CF clinic director, BC Children's Hospital. "CF Clinicians now have access to a life-changing therapy that targets this specific genetic mutation."
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