Regulatory Amylyx’s ALS drug Relyvrio receives FDA approval with a...

Amylyx’s ALS drug Relyvrio receives FDA approval with a 7-2 vote


Amylyx’s amyotrophic lateral sclerosis (ALS) drug, formerly known as AMX0035, has finally gained the approval of the Food and Drug Administration (FDA). The drug is presently marketed as Relyvrio and has been approved for treatment in adults.

Relyvrio had been under FDA review this past March where the advisory committee initially voted against it. The meeting ended with a negative 6-4 vote for the drug but Amylyx continued its efforts in developing the drug and seeking approval. Therefore, when the committee came together again this September, the company shared a new analysis with FDA which resulted in a positive 7-2 vote for the drug.

The drug is the first ALS treatment to show a significant deceleration in disease progression and functional decline. In a randomized clinical trial, the drug was also observed to extend survival. The approval for the drug can be credited to the phase 2 CENTAUR trial of the drug as well as the biomarker results of the drug in the phase 2 Alzheimer’s trial.

According to the co-founder of Amylyx, Justin Klee, it was initially thought that it would take trials at least till 2024 to obtain the best possible results, and the members of the committee who voted against the drug also felt that the results of the phase 3 PHEONIX trial needed to be taken into consideration before approval is granted. Typically when a company is seeking FDA approval, they need to present either two large studies or one extremely convincing study in order to gain acceptance. Contrary to this, the approval for Relyvrio is based on the results of a small mid-stage trial. 

“ALS patients simply do not have the luxury of time, if the approval was pending till 2024, that would have meant that a whole generation of ALS patients would have lost their lives. It seems unfair to deny patients access to all the available options” said, Kleen.

Apart from Amylyx, the ALS Association was also invested in FDA’s verdict. The organization had invested $2.2 million in the research and development of the drug and had led a long-spanning advocacy campaign to get approval for the drug. In September, the organization also submitted over 50,000 signatures to the FDA calling for them to approve the drug.

ALS disease leads to the destruction of nerve cells in the body. It causes difficulties with basic life functions like walking, talking, and swallowing. Presently around 20,000 people in the U.S. are diagnosed with the disease. In defense of its decision to afford regulatory flexibility to the drug, FDA has cited the life-threatening nature of the disease as the cause.

It is to be noted that Relyvrio has already been approved in Canada and it is expected that it would be approved in Europe sometime in mid-2023. The pricing that the company proposed in Canada equated to $165,000 and after calculating the cost-effectiveness of the drug, its value was assumed to be somewhere around $9,100 and $30,700 per year. With the FDA’s approval of the drug, it is now assumed that insurance companies will cover the drug.  

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