Canada first to approve experimental treatment for ALS

Canada has become the first country to approve a new drug for ALS, a progressive neurological disease that causes loss of muscle control and eventual paralysis.

But while Health Canada has approved the drug, called Albrioza, with conditions, the US Food and Drug Administration says it is not yet convinced the drug is effective.

The delay has ALS advocates in the United States fearing that it could be months, if not years, before Americans can access treatment that aims to slow the progression of the disease and extend the life of the disease. a patient’s life. On average, life expectancy since the onset of symptoms is two to five years.

However, other medical experts in the United States say that the FDA review process exists for a reason and that it is important that the drug meets standards before it is available.

The differing policies between Canada and the United States raise questions about the two processes and about the balance between approving or delaying new drugs that have a proven safety profile but are garnering more efficacy data.

ALS stands for amyotrophic lateral sclerosis and is also known as Lou Gehrig’s disease. There are few treatments.

Conditions for Health Canada’s approval of the treatment include that the drugmaker, Amylyx Pharmaceuticals, Inc. of Massachusetts, provide the government with data from another ongoing trial and that the company continue planned or ongoing studies.

« We are very excited, this is a great thing for Canadians, » said Tammy Moore, CEO of the ALS Society of Canada.

« With these therapies, we anticipate a slowing of disease progression, » said Moore, who was also featured in a press release about the drugmaker’s approval.

Health Canada told the Star that Albrioza was approved on June 10 with a Notice of Compliance with Conditions (NOC/c for short) and that an expedited review of the drug began in September 2021. The conditions were issued because it There are « some gaps in the information » that Amylyx has provided and the company is obligated to provide more information as it is revealed by additional research.

This type of approval “is granted for a medicinal product presenting a promising clinical benefit and an acceptable safety profile. An AC/C grants clearance to market the drug, provided the company performs confirmatory studies to verify the drug’s clinical benefit,” Health Canada said in a statement.

« Health Canada has concluded that the benefits of ALBRIOZA outweigh the risks when used as intended. »

In the United States, the FDA reviewed the drug in March and decided that it could not yet approve it because it was not convinced that the results of the only study that Amylyx completed clearly indicated that the drug was working.

There are approximately 3,000 Canadians living with ALS. About 1,000 people die each year from the disease and another 1,000 are diagnosed each year.

Jeff Derby, a B.C. resident who has lived with ALS since 2018, who came forward as a spokesperson via Amylyx, said his participation in the Albrioza clinical trial and continued use of the drug has slowed the progression of the disease, because he is still able to drive, walk using a walker and maintain his independence.

« What this will do is help ALS patients stick around long enough to keep fighting, » he said.

Based on data from the Albrioza clinical trial, people treated with the drug « scored an average of 2.32 points higher on the ALSFRS-R over a 24-week period, which could potentially signify a substantial change in a person’s ability to perform activities such as walking. , climb stairs, or breathe on your own longer,” according to a press release from Amylyx. The ALSFRS-R is a scale to measure the functionality of ALS patients.

Dr. G. Caleb Alexander, an epidemiologist and professor at the Johns Hopkins Bloomberg School of Public Health, was one of the members of the independent FDA advisory committee that voted earlier this year that there were not enough evidence to approve the drug.

He told the Star that while there is a « huge unmet need » for patient treatment, regulatory standards are there to ensure the drug is effective.

Data from only one trial is available, and Amylyx’s efficacy data comes more from the part of the trial that was an open-label extension, meaning those who took a placebo during an earlier part of the trial had the option of knowingly taking the drugs later, Alexander said.

“Ultimately, there is clearly promising evidence in this packaging…that this product could be an important new therapy. But those are not the regulatory thresholds that we have,” he said. The FDA can approve a drug with just one study, but that study must be « persuasive » and include « clear » evidence, and it lacks features of the Amylyx trial, he said.

Although Health Canada was able to issue conditional approval, what is called « accelerated approval » in the United States is based on a « biomarker », he said. A biomarker includes defined, measurable characteristics such as blood pressure or an X-ray, to determine outcomes, rather than how a person feels or functions, which the Amylyx test measured.

(COVID-19 vaccines and other COVID-related treatments and devices were originally approved in the United States under what are called emergency use authorizations.)

Accelerated approval standards focus on targeting the mechanisms that characterize the disease, when in this case the biological mechanisms of ALS are not well understood. Amylyx did not seek expedited approval, Alexander said.

Justin Klee and Joshua Cohen, founders of Amylyx, told The Star that they are delighted the drug has been approved in Canada and the cost of the drug is still being negotiated.

Together with the FDA, they are encouraged that the body is committed to reviewing their data and they hope to continue to work closely with them, Klee said. But the ALS patient community’s joy at the approval is an indication that the need is urgent, he said.

« It’s natural to have a back-and-forth scientific process, but we strongly stand behind our data, » Cohen said.

Neil Thakur, the ALS Association’s U.S. mission chief, said he’s thrilled for Canadian ALS patients, but is concerned about how long the 30,000 ALS patients in the U.S. will have to wait.

If a drug is safe and there’s a chance it could help, it’s important to give patients a chance to live longer now, rather than waiting for more evidence, he said.

« We’ve looked at this data very carefully, and we believe it provides benefits for people living with ALS today, » Thakur said.

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