A few days ago, Canada became the first country in the world to approve an experimental treatment for amyotrophic lateral sclerosis (ALS), a deadly and paralyzing neurological disorder.

This is AMX0035, which will be marketed in Canada under the name of Albrioza.

The Canadian authority has carried out the approval on an experimental basis until the pharmaceutical company,

Amylyx Pharmaceuticals

, provides more evidence that the treatment actually works.

AMX0035 combines two existing drugs:

sodium phenylbutyrate

, a supplement to regulate liver enzymes, and

taurursodiol

, to treat a pediatric urea disorder, which could protect neurons by preventing dysfunction of two structures in cells: the mitochondria and the endoplasmic reticulum .

The Canadian authority (Health Canada) gave Albrioza the green light under the formula called Notice of Compliance Conditions

, which allows the approval of drugs that appear promising for serious diseases, but have incomplete evidence that they work.

The central condition the agency set is that Amylyx "verify the clinical benefit of this drug" with data from a phase 3 clinical trial that is underway

and is expected to conclude in 2024, according to agency documents sent to the company.

The company must also conduct additional pharmacological studies and provide periodic safety reports.

In addition, the agency's communication warns that "patients must be informed."

From the US,

the Food and Drug Administration (FDA) is evaluating the same therapy

.

Earlier this year the US agency found the therapy safe but said there was not enough evidence it was effective in helping patients live longer or slow the rate at which they lose functions such as muscle control, speak or breathe without assistance.

A committee of independent FDA advisors narrowly voted in March that the therapy was not ready for approval.

The US regulatory agency had been scheduled to issue a decision later this month, but has finally extended the deadline to September 29, saying it needed more time to review further analysis of the data submitted by the company.

Despite the fact that conditional approval has not been carried out in the US, many American patients are showing interest, some even trying to purchase the compound through the Internet.

As a general rule, it is illegal for Americans to import drugs that have not been approved in the US for personal use.

But the FDA website lists some exceptions

that could apply to Albrioza, even if the drug doesn't have serious safety concerns and is to treat a serious condition for which no effective therapy is available in the US.

ALS, also known as Lou Gehring's disease, is the third most common neurodegenerative disease after dementia and Parkinson's.

According to the Spanish Society of Neurology, each year around 700 people begin to develop symptoms of ALS.

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