WASHINGTON (AP) — Amid controversy over its treatment for amyotrophic lateral sclerosis (ALS), Amylyx Pharmaceuticals announced Friday that its drug Relyvrio did not show efficacy in a key study and may no longer be available on the U.S. market as a result.
The drug, initially approved by the Food and Drug Administration in September 2022, was surrounded by a passionate campaign from individuals affected by ALS, commonly known as Lou Gehrig’s disease. Despite recent findings, Amylyx is yet to confirm whether it will secure its commitment to pull the drug should it fail to prove beneficial.
A follow-up study was unable to demonstrate the drug’s ability to slow disease progression or improve muscle strength when compared to a placebo. Relyvrio, like several other medicines for fatal and progressive conditions, has faced scrutiny due to inconclusive evidence of its effectiveness.
Initially, FDA approval hinged on a smaller, earlier phase study which, along with internal skepticism from the FDA’s scientists, resulted in an outside panel originally voting against Relyvrio. However, they altered their stance following further patient appeals.
Amylyx indicated it would voluntarily discontinue sales if the subsequent larger study involving over 600 patients did not substantiate the drug’s efficacy—a statement that significantly influenced the FDA advisory committee’s eventual favorable recommendation.
The drug’s full approval complicates matters for the FDA regulators, as it does not provide a straightforward route to mandate its withdrawal.
Prior to the initial approval, the FDA was subjected to considerable lobbying from ALS patients, advocates, and lawmakers. Priced at $158,000 for an annual supply, Relyvrio is a combination of a liver disorder prescription drug and a dietary supplement from traditional Chinese medicine. Its commercial performance has been underwhelming.
In the wake of the study’s results, stock value for the Cambridge, Mass.-based pharmaceutical company plummeted by more than 83% on Friday morning.
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Frequently Asked Questions (FAQs)
- What is Relyvrio?
Relyvrio is a drug made by Amylyx Pharmaceuticals for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. - Why is Relyvrio controversial?
The drug has been controversial due to doubts about its effectiveness in treating ALS, despite having received FDA approval. - What was the result of the follow-up study on Relyvrio?
The larger follow-up study revealed that Relyvrio was not effective in slowing down the progression of ALS nor improving muscle strength compared with a placebo. - Did the FDA approve the drug?
Yes, the FDA granted full approval to Relyvrio in September 2022. - What might happen to Relyvrio following the study?
Following the results of the follow-up study, Amylyx indicated that Relyvrio “may include voluntarily withdrawing” the drug from the market. - How was Relyvrio priced?
The drug was marketed at $158,000 for a year’s supply.
Conclusion
The latest developments regarding Relyvrio represent a disheartening setback for patients suffering from ALS who had placed their hopes in this treatment. Amylyx Pharmaceuticals’ consideration to possibly withdraw the drug from the market adheres to their initial pledge but simultaneously underlines the complexities of drug approval and the ever-present need for substantive, reliable efficacy data in the realm of life-threatening diseases. Patients, healthcare providers, and stakeholders in the ALS community will be closely monitoring the FDA’s next steps following this announcement.