First Ever Treatment For Morquio A Syndrome; Advocacy Group Calls For Expedited Access For Patients Throughout Canada
CANADA (July 7, 2014) – Health Canada has announced approval of Vimizim, a new enzyme replacement therapy (ERT) to treat Morquio A syndrome, a rare, devastating, and progressive disease that affects 70-100 people in Canada. Vimizim is manufactured by Biomarin Pharmaceuticals (BMRN) and is the first ever treatment available for this rare disorder.
Sufferers of Morquio A syndrome, or MPS IVA, lack an enzyme in their blood that breaks down cellular waste in the body called glycosaminoglycan (GAG). These GAGs build up in the bones, tissues, organs, and muscles of affected individuals and lead to many devastating symptoms including heart and airway disease, corneal clouding, skeletal abnormalities, decreased endurance, shortened stature, and premature death.
Andrew McFadyen, executive director of The Isaac Foundation and father of a 10 year old boy who suffers from a similar condition called MPS VI, says today’s decision provides hope for sufferers of Morquio A syndrome and their families. “Approval of this treatment is a huge step forward for the Morquio community, and provides hope for a promising future for these patients. Vimizim is the first treatment ever for this condition, and targets the underlying causes of the disease. As a whole, this will have a dramatic impact on the lives of sufferers. This is such incredible news.”
McFadyen notes that clinical trials were so effective that the FDA requested a priority review for approval, which was granted in February in the United States and in April in the European Union. Health Canada followed suit and requested a priority submission from Biomarin.
The approval means “new hope” for Debbie Braun, who lives in Ontario with her two boys, Lucas, 17, and Zane, 14. Both children suffer from Morquio A syndrome. “This has given our family the chance to think a little further into the boys’ future and to dream again of all the things they will be able to do and accomplish. It’s given us back the hope that was taken from us so long ago when we were diagnosed!”
Both Lucas and Zane have been part of the clinical trials for Vimizim and travel 6 hours round-trip to The Hospital for Sick Children in Toronto for their weekly infusions. Zane says it’s all been worth it, “I feel better on this drug and now all the other kids that have Morquio A syndrome can too.”
This sentiment is echoed by Lucas, who added his other reasons for participating in the clinical trial. “I did this so that other kids just like me could have this drug. I’m so happy that some of my friends can now go on this treatment. I feel so much better and I feel like I can do anything, and the first thing I want to do is learn how to drive!”
McFadyen is calling on all provincial governments to act quickly to ensure that reimbursement for the new treatment is forthcoming so that all patients in Canada can begin treatment immediately. He points out how important early diagnosis and treatment are in winning the battle against the disease. “With other enzyme replacement therapies, including the one my own son has been receiving for the past 8 years, we know that it can slow down or even halt the disease progression. It’s very important to begin treatment as soon as possible after diagnosis so that some of the devastating symptoms can be prevented, slowed down, or minimized. My son’s life was saved when he began ERT. With the incredible results seen during the clinical trials for Vimizim, I have every reason to believe this treatment will have the same results.”
Gaining access to the drug may prove difficult for families, as provincial governments have been notoriously slow to provide reimbursement so that treatments can begin in a timely fashion. “Part of the issue with providing access to rare disease treatments lies with the Federal government and our lack of an orphan drug policy in this country. But that’s only part of the problem. Provinces need to make the path clearer and easier, with evaluations on the effectiveness of rare disease treatments receiving a heightened priority as soon as they are approved by Health Canada. The current wait of two years or more is having a detrimental effect on patients who require immediate access to therapy.”
Currently, provinces require new treatments to undergo a Common Drug Review (CDR) before they even consider reimbursement and listing on provincial drug plans. This process typically takes 18 months or more. Once a CDR is completed, the drug in question heads to the Pan-Canadian Pricing Alliance (PCPA) so that negotiations can begin to achieve a reduced price from the drug manufacturer, a process that could take an additional 6 months to a year to complete.
“From an access standpoint, there is no reason for delay, and no reason why patients who need access can’t receive it soon after regulatory approval from Health Canada. Patients suffering from these rare and progressive diseases don’t have the luxury of time on their side, and the current process is long and arduous. As an advocate, and as a father of a child suffering from a rare disease, the process is frustrating. Gaining access to life-sustaining treatments is rarely a problem in most of the developed world but in Canada, denial of access is the norm.”
Debbie Braun agrees and hopes the provincial governments in Canada will allow patients to have immediate access to the new treatment. “I truly hope that all newly diagnosed patients with Morquio A syndrome can have access to this treatment before the disease has a chance to take from them what it has taken from my boys.”
Adds McFadyen, “These patients have a new hope of a healthier future, and I’m confident the provinces will work together and in an expedited fashion to see that hope become reality.”