Amylyx Pharmaceuticals Withdraws ALS Drug After Failed Clinical Trial

Amylyx Pharmaceuticals has announced a 70% reduction in its workforce and the voluntary withdrawal of its ALS medication, Relyvrio/Albrioza, following a clinical trial revealing its ineffectiveness. This move comes just after the US Food and Drug Administration's controversial approval of the drug based on a single trial. The company has initiated the removal process with regulators and will provide free drug access to existing patients during the transition. The decision is unusual, as drug removal from the market is typically uncommon.

Amylyx Pharmaceuticals Withdraws ALS Drug from Market

In a significant development, Amylyx Pharmaceuticals has announced its decision to voluntarily remove its drug, Relyvrio (Albrioza in Canada), from the market. This move comes less than a month after the release of a clinical trial demonstrating the inefficacy of the once-promising treatment for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease.

The Cambridge, Massachusetts-based company initiated the process of withdrawing the drug with regulators on Thursday. Effective immediately, Relyvrio will no longer be available for new patients. However, existing patients will have the option to transition to a "free drug program." Amylyx will also implement a 70% reduction in its workforce.

"While this is a challenging time for the ALS community, we reached this path forward in collaboration with affected stakeholders and in keeping with our unwavering commitment to individuals living with ALS and other neurodegenerative diseases," stated Joshua Cohen and Justin Klee, co-chief executive officers of Amylyx, in a joint statement.

The U.S. Food and Drug Administration's (FDA) approval of Relyvrio in 2022 sparked controversy due to its reliance on a single clinical trial. FDA staff and external experts raised concerns about the drug's effectiveness. A larger trial, the results of which were unveiled last month, revealed that the benefit to patients was not even as reliable as a coin toss. The company's stock value plummeted by 80% as a result.

Approximately 6,000 individuals in the United States are diagnosed with ALS annually. The progressive disease gradually impairs the body's ability to move, speak, and eventually breathe, with no known cure.

Amylyx's decision to voluntarily withdraw Relyvrio from the market is an uncommon occurrence. At an FDA meeting in 2022, an expert panel member expressed skepticism about the prospect of removing the drug from the market if it proved ineffective, suggesting that the FDA had underestimated the complexity and difficulty of such an action.

"Frankly, I'm not sure it's ever taken place, although admittedly in rare cases, manufacturers themselves have made the decision to do so," commented Caleb Alexander, an internist at Johns Hopkins University.

Alexander commended Amylyx's decision on Thursday. "This case also serves as a reminder of our standards for market access and the risks associated with diluting these standards based on factors other than scientific evidence," he emphasized.

The ALS Association similarly applauded the announcement. "We commend Amylyx for withdrawing Relyvrio from the market while ensuring that people living with ALS can still access the drug if they believe it is beneficial," the organization stated.

Amylyx, founded by Klee and Cohen while they were undergraduates at Brown University, had achieved significant success. The company gained FDA approval in 2022 based on a single trial indicating that Relyvrio slowed the rate of decline in activities such as walking and talking compared to a placebo. Subsequent data analysis also suggested that participants taking the drug lived an average of 10 months longer than a control group.

Relyvrio generated approximately $380 million in revenue last year. At its peak, Amylyx boasted a market capitalization exceeding $1 billion.

Typically, the FDA requires more substantial evidence—two confirmatory trials—before approving a drug. However, the agency sometimes exercises flexibility, considering the severity of the disease and the efficacy of existing treatments.

Amylyx announced its intention to continue analyzing the results of its failed larger trial to inform future ALS research. The company is also developing an additional ALS drug, but its primary drug candidate currently targets Wolfram syndrome, a hereditary disorder that leads to diabetes.

Correction:

An earlier version of this article incorrectly stated that Amylyx Pharmaceuticals made its announcement on Wednesday. The news was announced Thursday.