Maker Is Pulling Controversial ALS Drug Relyvrio Off the Market

April 4, 2024
Ernie Mundell HealthDay Reporter

THURSDAY, April 4, 2024 (HealthDayNews) -- Following disappointing trial results, the maker of a controversial ALS drug said it is pulling the medication off the market.

In a statement issued Thursday, Amylyx Pharmaceuticals said that Relyvrio failed to help patients in a large follow-up study, and the drug "will no longer be available for new patients as of today." The medication is sold as Albrioza in Canada.

"The decision to remove RELYVRIO/ALBRIOZA from the market and provide therapy free of charge for those who wish to continue was informed by the PHOENIX trial results, engagement with regulatory authorities and discussions with the ALS community," Joshua Cohen and Justin Klee, co-CEOs of Amylyx, said in the statement. "Thank you to each and every person who shared feedback with us and continues to support our commitment to the ALS community."

The company added that, as of Thursday, "patients currently on therapy in the U.S. and Canada who, in consultation with their physician, wish to continue can be transitioned to a free drug program."

Relyvrio combines two older drugs: a prescription medication for liver disorders and a dietary supplement associated with traditional Chinese medicine.

According to the New York Times, about 4,000 patients battling ALS are currently using Relyvrio, which has a list price of $154,000 per year.

Relyvrio was first approved by the U.S. Food and Drug Administration in September 2022, following a lengthy, impassioned campaign by patients with ALS, a fatal muscle-wasting disease.

Unfortunately, the latest study, dubbed PHOENIX, showed the drug did not slow the disease compared with a placebo treatment, and it also did not produce improvements on any secondary measures, such as muscle strength.

ALS is a devastating neurological disease that slowly destroys nerve cells and connections that are needed to walk, talk, speak and breathe. Most patients die within three to five years of their diagnosis.

Relyvrio's approval was mainly based on results from one small study that was criticized by some of the FDA's scientists. An outside committee of experts also voted against the drug initially, before being swayed to back it at a follow-up meeting requested by patients. At the time, Amylyx noted it was continuing a larger follow-up study of more than 600 patients that would provide further data on the drug.

In a highly unusual move, Amylyx executives at that second advisory committee meeting told FDA regulators they would pull the drug from the market if follow-up research didn't confirm its efficacy. That commitment seemed to reassure FDA's advisers, who then voted in favor of the drug's approval, despite the questionable data.

More information

The ALS Association has more on ALS.

SOURCE: Amylyx Pharmaceuticals, news release, April 4, 2024; New York Times

What This Means for You

The maker of a controversial ALS drug has pulled the medication from the market following lackluster results from a large trial.

Neurology Food &, Drug Administration