Could the Approval of Albrioza by Health Canada for ALS Impact the FDA Ruling?

This week, Health Canada announced its approval of Albrioza (created by Amylyx Pharmaceuticals) for the treatment of ALS. It has been the only newly approved treatment for ALS since 2018. The drug is also scheduled to be reviewed for FDA approval in the United States.  

ALS is a neurodegenerative disorder that impacts motor nerve cell function, causing muscle fatigue and paralysis. Symptoms of ALS can severely impair quality of life and may be fatal as difficulty breathing can occur. With 5,000 new ALS diagnoses annually in the US alone, researchers are constantly looking for ways to address symptoms and improve quality of life.  

Albrioza is composed of sodium phenylbutyrate and ursodoxicoltaurine. The Health Canada approval was based on a study published in the BMJ Journal of Neurology, Neurosurgery, and Psychiatry. This study used ALS patients within the first 18 months of symptom onset to test the efficacy of this drug combination.  

Members of the study were either given the sodium phenylbutyrate and ursodoxicoltaurine combination (at a ratio of 3:1 g) or a placebo. Over the span of 30 months, the patients were evaluated on the rates of key events they experienced, including tracheostomy, death, hospitalizations, and several others. 

The study concluded that patients who took the drug experienced 47% fewer key events than the placebo group. 

Amylyx’s website claims that Albrioza “may reduce neuronal cell death, hypothesized to occur by simultaneously mitigating endoplasmic reticulum (ER) stress and mitochondrial dysfunction.” 

If Albrioza continues to have a positive impact on ALS patients, it has the potential to optimize care and enhance the quality of life for ALS patients.  

This recent approval by Health Canada suggests the potential for FDA approval of Albrioza for ALS treatment. Amylyx published a statement in March of 2022 indicating that the FDA advisory board was split on the efficacy of Albrioza as an ALS medication, with the majority voting that it was not effective.  

While the FDA heavily weighs the opinions of its advisory board, the vote is not the final say on drug approval. The New Drug Application is still under review by the FDA, which is expected to decide by September 29, 2022. 

While Canadians begin to see the distribution and use of Albrioza, ALS patients, caretakers, and healthcare providers in the US will continue to monitor the FDA decision on Albrioza. If the FDA approves the drug, patients and providers will have to decide how it will impact current methods of care for ALS.  

Editors Note: This article was corrected on June 21, 2022 to reflect that the FDA was originally voted on the efficacy of Albrioza and will make their final decision in September.