Amylyx notches first global approval for oral ALS therapy Albrioza

Amylyx Pharmaceuticals (Nasdaq:AML) has received Health Canada approval for Albrioza (AMX0035).

The FDA is anticipated to decide on Albrioza, which consists of sodium phenylbutyrate and ursodoxicoltaurine, by September 29, 2022.

Amylyx is the first new therapy for ALS to win approval in Canada since 2018.

The approval also is the first global regulatory approval for the drug.

Health Canada decided to greenlight the drug after reviewing clinical trial data showing that it slowed disease progression and loss of functional decline in ALS patients.

“We are excited with Health Canada’s decision to approve Albrioza with conditions. Albrioza is a therapy that demonstrated in our CENTAUR trial a statistically significant and clinically meaningful impact on function, alone or in addition to existing ALS therapies,” said Justin Klee and Joshua Cohen, co-CEOs and co-founders of Amylyx, in prepared remarks. “We are grateful to the people who participat…

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Amylyx Pharmaceuticals preps NDA filing for ALS drug AMX0035  

Cambridge, Massachusetts–based Amylyx Pharmaceuticals intends to submit a New Drug Application (NDA) to FDA for AMX0035 (sodium phenylbutyrate and taurursodiol) to treat amyotrophic lateral sclerosis (ALS), which is also known as Lou Gehrig’s disease. 

The company decided to file after a series of meetings with the FDA, including a pre-NDA meeting on July 15.  

AMX0035 was the subject of the Phase 2 CENTAUR trial, which involved 137 participants with ALS. In that trial, the drug met its primary efficacy endpoint of slowing ALS based on the ALS Functional Rating Scale-Revised (ALSFRS-R). 

A forthcoming Phase 3 trial will evaluate the safety and efficacy of the drug over 48 weeks. 

Before reversing course, FDA had asked Amylyx to submit positive Phase 3 trial results before filing a New Drug Application.

To date, there are limited treatment options available for ALS. A 2020 paper in Neuropharmacology. 

Concluded that the two drugs with…

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