Health Canada has approved a breakthrough new treatment for Friedreich’s ataxia – a rare, degenerative and debilitating neurological disorder that attacks the nervous system and heart and makes walking difficult.
The company that manufactured Skyclarys, Biogen Canada Inc., said that that it is the only treatment in Canada to specifically target the underlying mechanism of the genetic disease.
Montreal neurologist Dr. Massimo Pandolfo was the first to detect the gene decades ago, and he said the new oral treatment offers a two-year pause in the illness.
“Friedreich’s ataxia presents profound physical challenges, progressively affecting motor function, independence, and overall quality of life,” Pandolfo said.
“As a physician and researcher in this field, I have seen firsthand the critical need for treatments that address the underlying causes of this devastating disease.”
Pandolfo explained that patients with Friedreich’s ataxia accumulate excess iron in their mitochondria, causing them to essentially “rust” and become toxic to cells. He said the treatment works by activating the cells’ natural defenses against the damage.
The illness affects approximately 1 in 40,000 people Canada, with an estimated 300 to 750 people currently living in the condition.
“The approval of Skyclarys is a significant advancement, offering the first therapy to go beyond managing symptoms and to targeting disease progression,” Pandolfo said.
“While not a cure, this development represents meaningful progress in treatment options and brings renewed hope to patients and their families.”
According to Biogen Canada, Friedreich’s ataxia causes progressive damage to the spinal cord, peripheral nerves, and the brain regions that control balance, movement, and speech.
Symptoms typically start in childhood or adolescence and worsen over time, leading to the loss of muscle coordination, speech and swallowing, and mobility, among other complications.
The average life expectancy for individual living with the disease is 37 years.
