Canada approves ERT Elfabrio for adults with Fabry disease
Clearance follows clinical trials showing treatment was safe, effective
Health Canada has approved the enzyme replacement therapy Elfabrio (pegunigalsidase alfa) to treat adults with Fabry disease.
“With Health Canada’s approval of Elfabrio, we’re proud to have a new treatment option available to Canadians living with Fabry disease,” John Hess, senior vice president for the Americas at Chiesi Global Rare Diseases, the therapy’s maker, said in a company press release. “This milestone reflects years of research and collaboration across the global rare disease community. We’ve seen the impact Elfabrio can have on patients worldwide and are pleased at the opportunity to expand access to Canadians.”
Fabry advocates in Canada welcomed the approval.
“While existing treatments have transformed care for many patients with Fabry disease, there remains a clear need for additional safe and effective treatments to treat Fabry Disease,” said Michael West, MD, director of the Nova Scotia Fabry disease program at QE II Health Sciences Center in Halifax. “The approval of Elfabrio, supported by clinical data and real-world experience, gives clinicians another option to address the varied needs of this patient community.”
Julia Alton, executive director of the Canadian Fabry Association, called the approval “a powerful moment for the Fabry disease community.”
“Having Elfabrio available in Canada offers families a new sense of momentum,” Alton said. “This kind of progress reminds us how important it is to continue lifting the voices of patients and caregivers who push for greater recognition, support, and understanding.”
Canadian approval follows nods in US, Europe
Fabry disease is caused by mutations in the gene that encodes alpha-galactosidase A, an enzyme necessary for breaking down certain fatty molecules. Without a functional version of this enzyme, these molecules accumulate to toxic levels in cells, leading to organ damage that ultimately drives Fabry disease symptoms.
Enzyme replacement therapies (ERTs) like Elfabrio are designed to deliver a healthy version of the enzyme to the body, allowing these fatty molecules to be broken down and ultimately slowing disease progression. Elfabrio is given by infusion into the bloodstream every other week. The medication has been approved for adults with Fabry disease in the U.S. as well as the European Union and the U.K.
The approvals were based mainly on data from three Phase 3 clinical trials — BRIDGE trial (NCT03018730), BALANCE (NCT02795676), and BRIGHT trial (NCT03180840) — that collectively included more than 140 adults with Fabry disease. Clinical data showed that Elfabrio is as effective as other ERTs at maintaining organ function. According to the Canadian prescribing information, common side effects observed in clinical trials include the common cold, respiratory infections, fatigue, headache, back pain in the back or extremities, cough, diarrhea, nausea, and fever.
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