Canada has approved Amylyx Pharmaceuticals’ treatment for amyotrophic lateral sclerosis (ALS), a neurodegenerative disease, the drugmaker said Monday, the first such approval for the oral drug.
The decision was subject to certain conditions, including the release of data from its late-stage global study of approximately 600 patients, expected in 2024, and additional studies.
The approval for the drug, sold under the Albrioza brand in Canada, is based on a mid-stage study in 137 patients and a long-term follow-up period that showed it slowed the decline in physical function in patients.
ALS, the cause of which is largely unknown, leads to the breakdown of nerve cells in the brain and spinal cord, impairing physical function and leading to severe disability and death.
About 3,000 Canadians are currently living with ALS, with an average life expectancy of two to five years from the onset of symptoms, the company says.
The Health Canada approval follows a decision by the U.S. Food and Drug Administration, which is expected by September 29.
A panel of outside advisers to the US regulator voted 6-4 against approval of the drug for ALS, AMX0035, in March, saying clinical trial data had not shown it to be effective against the disease.
ALS, commonly known as Lou Gehrig’s disease, came into the limelight in 2014 due to the “Ice Bucket Challenge,” in which people filmed pouring ice-cold water over their heads to raise awareness.
Reporting by Leroy Leo in Bengaluru; Edited by Arun Koyyur
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