Health Canada has approved a new drug treatment for adults living with Fabry disease, a rare inherited disorder that can affect multiple organs and significantly impact quality of life.
Chiesi Global Rare Diseases announced Tuesday that Elfabrio (pegunigalsidase alfa) has been authorized for use in Canada as a long-term enzyme replacement therapy (ERT) for adult patients diagnosed with Fabry disease.
The approval allows Elfabrio to become available to Canadian patients following its use in 28 countries worldwide, including the United States, Europe, and parts of the Asia-Pacific region, through public reimbursement systems and named patient programs.
Fabry disease is a progressive genetic condition caused by a deficiency of the enzyme alpha-galactosidase A. Without adequate levels of the enzyme, fatty substances accumulate in cells throughout the body, leading to damage in organs such as the kidneys, heart, brain, nervous system, and skin.
According to Chiesi, symptoms can include fatigue, chronic pain, gastrointestinal problems, reduced ability to sweat, progressive kidney failure, heart complications, and an increased risk of stroke.
The disease affects an estimated one in 40,000 to one in 60,000 males worldwide, with symptoms often varying widely in females.
Health Canada’s decision was supported by a clinical development program involving more than 140 patients, some of whom were followed for nearly eight years. The studies included both patients new to enzyme replacement therapy and those who had previously received treatment.
In clinical trials, Elfabrio performed comparably to existing Fabry disease treatments in preserving kidney function over time, measured using estimated glomerular filtration rate (eGFR), a standard indicator of kidney health.
Most side effects reported during the studies were described as mild to moderate. However, Health Canada cautions that the drug should not be used in patients with known hypersensitivity to the medication or its ingredients. The company also noted that severe allergic reactions, including anaphylaxis, have been reported, and patients should be monitored during and after infusions with appropriate medical support available.
Fabry disease can present at any stage of life, from childhood through adulthood, and is often misdiagnosed or diagnosed late due to its broad range of symptoms. Chiesi emphasized that early detection and timely access to treatment are critical to slowing disease progression and managing long-term complications.