Health Canada approves drug treatment for rare disease

A patient visits a doctor. (Pexels)

Health Canada has approved a new drug treatment for adults living with Fabry disease, a rare genetic disorder that can affect multiple organs and impact the quality of life.

Chiesi Global Rare Diseases announced Tuesday in a press release that Elfabrio (pegunigalsidase alfa) has been authorized for use in Canada as a long-term enzyme replacement therapy (ERT) for adults diagnosed with Fabry disease.

The approval makes Elfabrio available to Canadian patients following its use in 28 countries worldwide through public reimbursement systems and named patient programs, including in the U.S., Europe and parts of the Asia-Pacific region.

According to the press release, Fabry is a progressive, inherited condition caused by a deficiency of the enzyme alpha-galactosidase A. Without sufficient levels of the enzyme, fatty substances build in cells throughout the body, damaging organs such as kidneys, heart, brain, nervous system and skin.

“Fabry disease can cause a range of serious signs and symptoms, including fatigue, chronic pain, gastrointestinal issues, decreased ability to sweat, progressive kidney failure, heart complications, and increased risk of stroke,” the press release read.

The disease affects an estimated one in 40,000 to one in 60,000 males globally, with symptoms that can vary widely in females, the biopharmaceutical group said.

According to the company, Health Canada’s decision was supported by a clinical development program involving more than 140 patients, with some followed for up to nearly eight years.

Studies included both patients new to enzyme replacement therapy and those previously treated.

In the studies, the company says Elfabrio worked about as well as any existing Fabry disease treatment at protecting kidney function over time.

Doctors measured this by tracking how well the kidneys filtered waste from the blood, a standard test known as eGFR.

Most side effects reported by patients were not severe with the company saying they were generally mild to moderate. Health Canada cautions that the drug is unsuitable in patients with hypersensitivity to the medication or its ingredients, Chiesi said.

They also warned 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 from childhood through adulthood and is often misdiagnosed or diagnosed late due to its wide range of symptoms, Chiesi said.

The company warned that early detection and access to appropriate treatments are critical for slowing disease progression and managing long-term complications.