Statement on Relyvrio Clinical Trial Results

We are saddened by today’s news about Relyvrio and our hearts go out to everyone in the ALS community, which desperately needs new safe and effective treatments. We are learning this news with everyone else in real time so we hope to meet with Amylyx soon and learn more about the trial results. We thank everyone who participated in the PHOENIX trial.

We supported the early approval of Relyvrio because there were no questions about its safety and the phase 2 trial data was positive. At the time, we also called for an additional trial of Relyvrio to confirm its effectiveness. Had the FDA not approved early and then the PHOENIX trial results had been positive, thousands of people living with ALS would have not had access to a life-extending treatment that was safe for over two years. Our community expressed to the FDA that they were willing to take the risk if it turned out to be ineffective. Now that the confirmatory trial has failed, we hope Amylyx will consult with all stakeholders in the ALS community on next steps.

We are grateful to the FDA and Amylyx for working closely with the ALS community throughout this process and we believe there is still a lot to be gained from this experience. While today’s news is disappointing, there are more than 50 potential treatments in the clinical stage of development, including more than a dozen in phase 3 trials. We are more committed than ever to ensuring that safe and effective treatments are approved and available to people living with ALS as quickly as possible.

UPDATE – APRIL 2024: Relyvrio was voluntarily withdrawn from the market by Amylyx following a phase 3 trial that failed to show it was effective. The ALS Association stands by its decision to push for early approval of Relyvrio given the promising phase 2 trial data and the safety of the treatment. At the time, we said that if it turns out to be ineffective, at worst, people living with ALS would have taken an ineffective therapy without risk of harm. If it was indeed effective, delaying access would have meant that people living with ALS would have lost two years of being able to take a life-extending therapy. In the interests of transparency and education, we are leaving this information up for future reference. People living with ALS need life-saving treatments and we are working as urgently as possible to advance the many more potential treatments in clinical trials.