December 16,2025
RED FM News Desk
Health Canada has approved a new medication for adults with Fabry disease, a rare inherited disorder that can damage multiple organs and significantly affect quality of life.
The drug, Elfabrio (pegunigalsidase alfa), developed by Chiesi Global Rare Diseases, is now authorized in Canada as a long-term enzyme replacement therapy for adults diagnosed with the condition. The approval allows Canadian patients access to a treatment that is already available in 28 countries, including the United States, Europe and parts of the Asia-Pacific region.
Fabry disease is caused by a deficiency of the enzyme alpha-galactosidase A, which leads to the buildup of fatty substances in cells and can harm the kidneys, heart, brain, nervous system and skin. Symptoms may include chronic pain, fatigue, digestive problems, reduced sweating, kidney failure, heart disease and an increased risk of stroke.
Health Canada’s decision was supported by clinical studies involving more than 140 patients, some followed for nearly eight years. Research showed that Elfabrio was comparable to existing treatments in preserving kidney function, as measured by standard kidney filtration tests. Most reported side effects were mild to moderate, though the drug is not recommended for patients with known hypersensitivity and carries a risk of serious allergic reactions.
Chiesi emphasized that Fabry disease can be difficult to diagnose and may appear at different stages of life, making early detection and timely treatment essential to slowing disease progression and reducing long-term complications.
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