Canadians with the gene disorder Fabry disease -- many of whom have no access to drug therapy -- will now be able to obtain two staggeringly expensive medications under a new government deal. Fabry patients will enroll in a research study to receive medicine to treat the rare disorder, the Canadian government announced.
"Federal and provincial governments and two drug developers are about to enter into a three-year funding agreement to support an independent post-market study of enzyme replacement therapies for Fabry disease," said a draft patient fact-sheet from the federal and provincial governments that was obtained by the Canadian newspaper The Globe and Mail.
An estimated 5,000 people worldwide, including more than 200 people in Canada, have Fabry disease, which primarily afflicts men. Those who inherit the abnormal gene cannot produce enough of a particular fat-eating enzyme. They typically experience pain, difficulty sweating and skin rashes.
Although Health Canada has licensed two drugs to treat the condition -- Fabrazyme and Replagal -- provinces have struggled to pay for them. The drugs are typically very expensive -- for instance, it costs $275,000 to treat the average patient for a year with Fabrazyme. Under the new government deal, patients would be able to get treatment so long as they become part of a research protocol. Specific details of the protocol were not reported.