Relyvrio Approved as ALS Treatment by the FDA

Amylyx Pharmaceuticals recently announced in a press release that the US Food and Drug Administration (FDA) approved Relyvrio for the treatment of adults with amyotrophic lateral sclerosis (ALS). Additionally, it has been approved with conditions as Albrioza for the treatment of ALS in Canada.

ALS is a rare, progressive neurodegenerative disease that causes death of motor neurons in the brain and spinal cord. Motor neurons control voluntary muscles that allow us to do things not usually given a second thought, such as chewing, breathing, talking and walking. As the motor neurons lose their ability to activate muscles, those muscles become weaker over time and eventually can lead to the inability to speak, move and respiratory paralysis. Respiratory failure is the primary cause of death, very shortly after symptoms first appear.

In the US alone, over 20,000 individuals are currently living with ALS, and more than 90 percent of people with ALS develop it sporadically with no family history. Although there is no cure for the disease, there are some treatments available that have been shown to improve life expectancy and reduce decline in function.

Relyvrio, formerly known as AMX0035 in the US, showed in clinical trials that it significantly slowed the loss of physical function in those with ALS.

Joshua Cohen and Justin Klee, Co-CEOs of Amylyx, stated that the “FDA approval of Relyvrio is an exciting milestone for the ALS community and is a major step toward achieving our mission to one day end the suffering caused by neurodegenerative diseases.”

Relyvrio (sodium phenylbutyrate and taurursodiol) is an oral, fixed dose medication that is taken by combining one packet of the medication with 8 ounces of room temperature water. Optionally, it can be administered through a feeding tube.

The recommended dose for the first three weeks is one packet daily, which consists of 3 grams sodium phenylbutyrate and 1 gram taurursodiol. After three weeks, the dose should be increased to one packet, twice daily. Relyvrio can be taken as monotherapy or in combination with currently approved treatments.

XTALKS WEBINAR: How Sponsors are Accelerating Clinical Trials with Automatic EHR Integration

Live and On-Demand: Friday, November 4, 2022, at 10am EDT (3pm CET/EU-Central)

Register for this free webinar to learn how to accelerate study timelines and decision-making through automated electronic health record (EHR) and electronic data capture (EDC) integration.

Investigation of Relyvrio in the CENTAUR Clinical Trial

The efficacy of Relyvrio was investigated in the CENTAUR trial, a multi-center, randomized, placebo-controlled Phase II clinical trial. The trial involved 137 adult participants between the ages of 18 to 80, currently diagnosed with ALS.

The primary efficacy endpoint was achieved at the end of the 24-week study, as Relyvrio significantly slowed the loss of physical function in study participants. Loss of physical function was measured with the ALS Functional Rating Scale, in which four subdomains of function (bulbar, fine motor, gross motor and breathing) are scored on a scale from 0 to 4, where 0 is a total loss of function and 4 is no loss of function, with higher scores indicating better function.

The study also included an open-label extension long-term follow-up phase to observe longer term effects of Relyvrio. The most common side effects associated with Relyvrio were predominantly gastrointestinal adverse reactions in the first three weeks of treatment, and upper respiratory tract infection.

RELATED: ALS Association Pressures FDA to Approve Amylyx Drug as Agency Cuts Phase III Trial Requirement

A Step Forward in ALS Care

Presently, the European Medicines Agency is reviewing Amylyx Pharmaceuticals’ Marketing Authorization Application for Relyvrio as a treatment option for ALS in Europe. Additionally, the drug is being investigated as a possible treatment for other neurodegenerative diseases.

To overcome barriers to access, Amylyx had made the decision to price the medication below the last FDA-approved ALS treatment available to patients. Amylyx will also support healthcare professionals, those living with ALS, and their families through the Amylyx Care Team (ACT) Support Program, which includes financial assistance options, education and other resources.

The approval of Relyvrio has garnered positive commentary from many US ALS advocacy groups, such as the ALS Association, Answer ALS Foundation, I AM ALS, Les Turner ALS Foundation and Team Gleason.

The organizations stated that “Relyvrio can offer people living with ALS and their families the potential of more time with functional independence. This is especially important for a rapidly progressive disease with a median survival time from diagnosis of just two to three years. This is significant for people living with ALS, their loved ones, caregivers, clinicians, researchers and advocacy, as we now have a new treatment option that could be a big step forward for the future of ALS care.”