Chiesi Global Rare Diseases Announces Health Canada Approval of Elfabrio® (pegunigalsidase alfa) for Fabry Disease

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Approval of Elfabrio® provides an additional treatment option for those living with Fabry disease

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PARMA, Italy and WOODBRIDGE, Ontario, Dec. 16, 2025 (GLOBE NEWSWIRE) — Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, today announced the Health Canada approval of Elfabrio^® (pegunigalsidase alfa) for the treatment of adults with Fabry disease. Elfabrio is commercially available in 28 countries worldwide through national and regional reimbursement pathways and Named Patient Programs (NPP), with broad geographic coverage across the US, Europe, and the Asia-Pacific region.

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Fabry disease is a rare, progressive, genetic condition that impacts an estimated 1 in 40,000 to 1 in 60,000 males worldwide, with variable expression in females.^i,ii Caused by a deficiency of the enzyme alpha-galactosidase A, Fabry disease leads to a buildup of certain lipids in cells throughout the body, affecting the skin, eyes, gastrointestinal system, kidneys, heart, brain, and nervous system.

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“With Health Canada’s approval of Elfabrio, we’re proud to have a new treatment option available to Canadians living with Fabry disease,” said John
Hess,
Senior Vice
President,
Americas,
Chiesi
Global
Rare
Diseases. “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. Our goal has always been to broaden what’s possible for patients and families impacted by rare diseases by bringing forward treatments that not only address the underlying disease but also improve the experience of care. We would like to thank the investigators, and, above all, the courage of the patients and families who made this advancement possible.”

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“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,
M.D.,
FRCPC,
FACP,
Director,
Nova Scotia
Fabry
Disease
Program,
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.”

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Julia
Alton,
Executive
Director,
Canadian
Fabry
Association, said, “Today’s approval is a powerful moment for the Fabry disease community. Having Elfabrio available in Canada offers families a new sense of momentum. 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.”

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Health Canada’s approval is supported by a comprehensive clinical development program, which evaluated the safety, tolerability, and efficacy of Elfabrio in more than 140 patients with up to 7.5 years of follow-up treatment. Elfabrio has been studied in both enzyme replacement therapy (ERT)-naïve and ERT-experienced patients, including a head-to-head trial that met its primary endpoint with Elfabrio demonstrating non-inferior efficacy to agalsidase beta in controlling estimated glomerular filtration rate (eGFR) decline, and in which Elfabrio was generally well-tolerated, with the majority of adverse events being mild or moderate in intensity.