Amylyx’s ALS drug gets conditional approval in Canada

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Amylyx’s ALS drug gets conditional approval in Canada

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Amylyx’s ALS drug gets conditional approval in Canada.

The first in the world! Amylyx’s ALS drug gets conditional approval in Canada, but FDA delays review.

A few days ago, Health Canada announced the conditional approval of Amylyx Pharmaceuticals’ amyotrophic lateral sclerosis (ALS) drug Albrioza (AMX0035), which is the company’s first approval in the world, and Albrioza has become the first approved ALS drugs in Canada.

Previously, Amylyx had faced some setbacks in its regulatory filings in the U.S. market. The drug received a pessimistic analysis after an internal FDA review, and an independent FDA advisory panel voted to reject the ALS treatment, with expert advisors arguing that Amylyx needs more trial data to prove Albrioza’s efficacy .

Ultimately, the U.S. Food and Drug Administration pushed the decision deadline for the therapy to September 29 this year.

Health Canada approved Albrioza based on data from a Phase 2 trial called CENTAUR and longer open-label follow-up. In the CENTAUR trial, researchers tested Albrioza versus placebo in 137 ALS patients for six months.

The results of the trial showed that patients who took the drug after 24 weeks had an average score of 2.32 points higher on the Amyotrophic Lateral Sclerosis Functional Rating Scale.

ALS is a serious neurodegenerative disease that typically has an average lifespan of two to five years after diagnosis.

Tammy Moore, CEO of the Canadian ALS Association, said in a statement that following regulatory approval, most importantly, all Canadians will be able to benefit from this innovative treatment.

Moore also called for regulatory decision makers to work swiftly throughout the drug access and reimbursement process to provide timely and equitable access to treatment for all Canadians who may benefit.

Albrioza contains two ingredients, sodium phenylbutrate (PB) and taurursodiol (TURSO).

Sodium phenylbutyrate is a small molecule protein chaperone that reduces the unfolded protein response (UPC) and prevents UPC-induced cell death. Taurine diol is a Bax protein inhibitor that improves mitochondrial and endoplasmic reticulum health in cells.

However, while Canadian regulators approved the drug, U.S. FDA staff faced the same trial data and did not find it particularly convincing.

FDA staff said Amylyx’s data analysis should include deaths that occur during the natural course of the disease, even if those deaths were not related to treatment.

However, the Canadian regulatory approval of the therapy is also conditional, for which Amylyx will have to submit data from its ongoing Phase 3 PHOENIX trial, which is expected to be released in 2024.

In addition to this, Amylyx said in its approval announcement that Canadian regulators are also requiring Amylyx to conduct “additional program plans and other ongoing studies.”

Reference source:

Amid FDA delay, Amylyx scores conditional ALS drug nod in Canada

Amylyx’s ALS drug gets conditional approval in Canada

(source:internet, reference only)

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