Novel ALS Drug – Relyvrio – Approved by FDA

Long awaited by the individuals and families impacted by amyotrophic lateral sclerosis (ALS) or Lou Gehrig’s disease, a progressive and often fatal rare disease that attacks and kills the nerve cells that control muscles needed for voluntary movements, Relyvrio™ was approved as a treatment by the FDA in September 2022. An oral, fixed-dose combination therapy, its ingredients apparently work together to prevent nerve cell death. Prior to this approval, only two other drugs -- Rilutek® (riluzole), and Radicava® (edaravone) -- were approved by the FDA to treat ALS although they provided a modest therapeutic benefit.

Approximately 25,000 people in the US live with ALS and most patients die of respiratory failure within 2 to 5 years of symptom onset.

Relyvrio is dosed once daily for the first 21 days and twice daily thereafter, at the wholesale acquisition cost (WAC) during the first year of treatment at $158,313 and $163,002 annually for continuous twice daily dosing. The September 2022 Institute of Clinical and Economic Review (ICER) summary on its cost-effectiveness stated that while evidence is adequate to demonstrate that Relyvrio provides a net health benefit compared to standard of care with riluzole, it would only achieve common thresholds for cost-effectiveness if priced between $9,100 and $30,700 per year -- a “low” long-term value for the price.

Relyvrio appears to be safe and well tolerated, providing a new mechanism of action to assist in the treatment of ALS patients.

To learn more about Relyvrio, click here.