Health Canada Approves Elfabrio for Fabry Disease Treatment, Expanding Access to PEGylated Enzyme Replacement Therapy
Key Insights
Health Canada has approved Elfabrio (pegunigalsidase alfa), a PEGylated enzyme replacement therapy for treating adults with Fabry disease (search), marking the drug's availability in its 29th country worldwide.
The approval is supported by comprehensive clinical data from over 140 patients with up to 7.5 years of follow-up, demonstrating non-inferior efficacy to agalsidase beta in controlling kidney function decline.
Fabry disease (search) affects an estimated 1 in 40,000 to 1 in 60,000 males worldwide and is caused by alpha-galactosidase A (search) enzyme deficiency, leading to progressive multi-organ complications.
Chiesi Global Rare Diseases (search) announced Health Canada's approval of Elfabrio (pegunigalsidase alfa) for treating adults with Fabry disease (search) on December 16, 2025. This regulatory milestone expands access to the PEGylated enzyme replacement therapy to Canadian patients, bringing the total number of countries where Elfabrio is commercially available to 29 through national and regional reimbursement pathways and Named Patient Programs.
The approval addresses a significant unmet medical need for patients with Fabry disease (search), a rare genetic condition affecting an estimated 1 in 40,000 to 1 in 60,000 males worldwide, with variable expression in females. The disease results from mutations in the GLA gene (search), causing deficiency of the enzyme alpha-galactosidase A (search) and subsequent accumulation of globotriaosylceramide (GL-3) in cells throughout the body.
Clinical Evidence Supporting Approval
Health Canada's decision is backed by a comprehensive clinical development program that evaluated Elfabrio's safety, tolerability, and efficacy in more than 140 patients with up to 7.5 years of follow-up treatment. The studies included both enzyme replacement therapy-naïve and ERT-experienced patients.
A pivotal head-to-head trial demonstrated that Elfabrio met its primary endpoint, showing non-inferior efficacy to agalsidase beta in controlling estimated glomerular filtration rate (eGFR) decline. The treatment was generally well-tolerated, with the majority of adverse events being mild or moderate in intensity.
"While existing treatments have transformed care for many patients with Fabry disease (search), there remains a clear need for additional safe and effective treatments to treat Fabry Disease," said Michael West, M.D., FRCPC, FACP, Director of the Nova Scotia Fabry Disease Program at QE II Health Sciences Centre, 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."
Therapeutic Innovation and Mechanism
Elfabrio represents a technological advancement in enzyme replacement therapy through its PEGylated formulation. The drug is a plant cell culture-expressed, chemically modified stabilized recombinant version of the α-Galactosidase-A enzyme. Protein sub-units are covalently bound via chemical cross-linking using short PEG moieties, resulting in stable pharmacokinetic parameters.
In clinical studies, Elfabrio demonstrated an initial half-life of 78.9 ± 10.3 hours. However, clinical studies have not established that this extended half-life results in superior efficacy or safety based on clinically relevant endpoints.
Disease Impact and Treatment Landscape
Fabry disease (search) manifests as a progressive, multi-system disorder affecting the heart, kidneys, skin, nervous system, and other organs. Patients experience a range of serious symptoms including fatigue, chronic pain, gastrointestinal issues, decreased ability to sweat, progressive kidney failure, heart complications, and increased stroke risk.
The condition can present from childhood through adulthood and is often subject to delayed diagnosis or misdiagnosis. Early detection and access to appropriate treatment, such as enzyme replacement therapy or pharmacological chaperones, are critical for managing symptoms and slowing disease progression.
Safety Profile and Monitoring Requirements
Elfabrio carries important safety considerations, particularly regarding hypersensitivity reactions including anaphylaxis. Patients treated with the therapy have experienced severe hypersensitivity reactions, necessitating that appropriate medical support measures, including cardiopulmonary resuscitation equipment, be readily available during administration.
The drug is contraindicated in patients who are hypersensitive to pegunigalsidase alfa or any ingredient in the formulation. Healthcare providers must monitor patients during and after infusions, and if severe hypersensitivity reactions occur, infusion should be immediately paused with appropriate medical treatment initiated.
Industry and Patient Community Response
John Hess, Senior Vice President, Americas, Chiesi Global Rare Diseases (search), emphasized the significance of the approval: "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."
Julia Alton, Executive Director of the Canadian Fabry Association, described the approval as "a powerful moment for the Fabry disease (search) community," noting that having Elfabrio available in Canada "offers families a new sense of momentum."
The approval strengthens Chiesi's position in the rare disease space, with Elfabrio now available across the US, Europe, and Asia-Pacific region. The company's Global Rare Diseases unit focuses on delivering innovative therapies and solutions for people living with rare diseases, collaborating with the global rare disease community to bring voice to underserved populations in the healthcare system.