Canada becomes the first country in the world to approve experimental therapy for amyotrophic lateral sclerosis (ALS), a deadly and crippling neurological disorder with few effective treatment options. AMX0035, to be marketed in the country under the name Albrioza, has been given the green light, but on the condition that Amylyx Pharmaceuticals, the small Massachusetts pharmaceutical company that produces it, provide more information on its effectiveness.
Every year around the world, 6,000 cases of ALS are diagnosed, a progressive pathology that kills people within two to five years and for which there is no cure. Today there are only two drugs indicated for this disease and they are approved in the United States, riluzole, which can extend the patient’s survival by several months, and edaravone, capable of slowing down the progression of degeneration by 33%.
This new therapy, Albrioza is a drug that combines two compounds: taurursodiol, a supplement often used to regulate liver enzymes, and sodium phenylbutyrate, a drug to treat a pediatric urea disorder that could protect neurons by preventing dysfunction of mitochondria and the endoplasmic reticulum.
The format of this medicine approved by Canada is a powder, with a bitter taste and that must be mixed with water to be drunk or inserted through a tube, depending on the patient’s condition, twice a day.
Albrioza slows down functional deterioration by 25%
Testing so far in a phase 2 trial indicates that Albrioza was able to slow the functional decline of participants by 25% compared to those given placebo, a decrease of 2.32 points less on a 48-point scale in ELA, which rates 12 physical skills, such as walking, talking, swallowing, dressing, handwriting, and breathing.
“Health Canada concluded that the benefits of Albrioza outweigh the risks when used as intended”
Patients who received the treatment for the longest time had about 6.5 months longer before being hospitalized, put on a ventilator or dying than those who did not take this medication. Although they seem very encouraging, these results do not seem to be enough for the United States Food and Drug Administration (FDA), which is why earlier this year it declared that the therapy was not yet ready for approval and that the final decision They would give a maximum of September 29 after reviewing all the data presented by the company.
And it is that, the FDA generally needs two clinical trials of a drug so that it can be approved, however, in cases of serious illness for which there are few treatments, the organization can consider the evidence of a clinical trial added to supporting data additional. Other countries such as Argentina are also considering its approval.
Health Canada, the authority equivalent to the FDA in this country, has given the drug the green light, but on the condition that the pharmaceutical company verify the clinical benefit of this drug, to do so a phase 3 clinical trial is already underway that could conclude in the year 2024. In addition, they must present additional pharmacological analyzes on the safety of this compound.
“Canadians living with ALS have limited options for their treatment. Following a thorough review of the information provided by the company in its drug filing, Health Canada concluded that the benefits of Albrioza outweigh the risks when used as intended.
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