Canada is the first nation to approve Albrioza, an experimental ALS medication
Key Takeaways:
- An experimental treatment for amyotrophic lateral sclerosis, also known as Lou Gehrig's disease, has been approved by Health Canada.
- After delaying its decision by three months to allow more time for more research from Amylyx's clinical studies to be examined, the FDA is slated to make its decision on Sept. 29.
- The ALS Society of Canada has urged decision-makers to act quickly to ensure that commercial and public drug plans cover Albrioza.
Despite ongoing doubts regarding the medicine's therapeutic benefit, Health Canada has approved an experimental treatment for amyotrophic lateral sclerosis, better known as ALS or Lou Gehrig's disease.
In a news release issued Monday, Amylyx Pharmaceuticals stated that Canada is the first jurisdiction to approve Albrioza as a treatment for the terrible neurological disease.
Health Canada issued its approval under a strategy to accelerate market access for promising drugs for critical disorders, with the Massachusetts-based maker agreeing to provide further data and research.
The Food and Drug Administration in the US has declared it safe. However, it is still reviewing Albrioza after an independent advisory group narrowly decided earlier this year that current evidence on the drug's effectiveness did not meet the bar for approval. The FDA is set to make its decision on Sept. 29 after delaying it by three months to enable more time for additional analysis from Amylyx's clinical studies to be reviewed.
According to the ALS Society of Canada, 3,000 Canadians suffer from incurable motor neuron disease, which causes paralysis, inability to swallow, respiratory failure, and death within two to five years of onset of symptoms.
Amylyx claims that Albrioza, a powdered combination of sodium phenylbutyrate and taurursodiol, can prevent motor neuron death by preventing damage and malfunction in two essential components of the cell.
US and Canadian regulators evaluated data from a clinical trial involving 137 people with ALS. This included a six-month randomized, controlled phase in which Albrioza was compared to a placebo and follow-up research in which patients were aware they were taking the treatment. More research is being carried out.
On a 48-point ALS scale, which employs a questionnaire to determine how the disease affects a person's bodily functions, participants who took Albrioza scored 2.3 points higher on average.
Albrioza is expected to be commercially available in Canada in about six weeks, according to Amylyx. Still, the firm is not announcing the price while going through the public drug-pricing assessment process.
The ALS Society of Canada has encouraged decision-makers to move fast to ensure that private and public drug plans cover Albrioza.