FDA-approved ALS drug Relyvrio could be pulled from market after failing clinical trial
One of the few available drugs to treat amyotrophic lateral sclerosis, or ALS, may get pulled from the market after failing a large clinical trial nearly two years after its controversial approval by the Food and Drug Administration.
The Phase 3 trial investigators found “no significant difference” in patients who took the drug, called Relyvrio, compared with patients who took a placebo, according to a statement released Friday by the drug’s manufacturer Amylyx Pharmaceuticals.
In the 48-week trial, the Cambridge, Massachusetts-based company enrolled 664 adults living with ALS. The disease, also commonly known as Lou Gehrig's disease, gradually destroys nerve connections needed for basic movements and – eventually – breathing. There is no cure and most people die within three to five years of diagnosis.
Justin Klee and Joshua Cohen, who are co-CEOs of Amylyx, said the company will continue to work with federal regulators and the broader ALS community to discuss results from the trial and decide about Relyvrio's fate in the next eight weeks. They may voluntarily withdraw the drug from the market, they said. In the meantime, the company has decided to pause promotion.
“We are surprised and deeply disappointed by the (trial) results,” Klee and Cohen said. “We will be led in our decisions by two key principles: doing what is right for people living with ALS, informed by regulatory authorities and the ALS community, and by what the science tells us.”
Relyvrio, a combination of two drugs, sodium phenylbutyrate and taurursodiol, was supposed to work by boosting cell energy and preventing a process that can lead to cell death. It was taken orally or through a feeding tube.
Amid pressure from patients unwilling and unable to wait for a years-long review process, the drug received full FDA approval after a small, mid-stage study in which patients appeared to progress more slowly and survive several months longer. Normally, the agency requires two large studies or one study suggesting a "very persuasive" improvement in survival.
Several other drugs have been approved to treat ALS, the most recent in 2017, but none has been shown to stop the loss of muscle control. Only one has been shown to extend life in some people, but by just three months on average.
Contributing: Karen Weintraub, Paste BN; Associated Press.
Adrianna Rodriguez is a health reporter for the Paste BN nation team. Contact Adrianna at adrodriguez@usatoday.com or @AdriannaUSAT on X.