$158,000 ALS drug pulled from market after failing in large clinical trial

Off the market —

The drug is now unavailable to new patients; its maker to lay off 70% of employees.

Beth Mole
– Apr 5, 2024 10:28 pm UTC

Amylyx, the maker of a new drug to treat ALS, is pulling that drug from the market and laying off 70 percent of its workers after a large clinical trial found that the drug did not help patients, according to an announcement from the company Thursday.

The drug, Relyvrio, won approval from the Food and Drug Administration in September 2022 to slow the progression of ALS (amyotrophic lateral sclerosis, or Lou Gehrig’s disease). However, the data behind the controversial decision was shaky at best; it was based on a study of just 137 patients that had several weaknesses and questionable statistical significance, and FDA advisors initially voted against approval. Still, given the severity of the neurogenerative disease and lack of effective treatments, the FDA ultimately granted approval under the condition that the company was working on a Phase III clinical trial to solidify its claimed benefits.

Relyvrio—a combination of two existing, generic drugs—went on the market with a list price of $158,000.

Last month, the company announced the top-line results from that 48-week, randomized, placebo-controlled trial involving 664 patients: Relyvrio failed to meet any of the trial’s goals. The drug did not improve patients’ physical functions, which were scored on a standardized ALS-specific test, nor did it improve quality of life, respiratory function, or overall survival. At that time, the co-CEOs of the company said they were “surprised and deeply disappointed” by the result, and the company acknowledged that it was considering voluntarily withdrawing the drug from the market.

In the announcement on Thursday, the company called Relyvrio’s market withdrawal a “difficult moment for the ALS community.” Patients already taking the medication who wish to continue taking it will be able to do so through a free drug program, the company said. It is no longer available to new patients, effective Thursday.

Amylyx is now “restructuring” to focus on two other drug candidates that treat different neurodegenerative disease. The change will include laying off 70 percent of its workforce, which, according to The Washington Post, includes more than 350 employees.

Relyvrio is part of a series of similarly controversial drugs for devastating neurodegenerative diseases that have gained FDA approval despite questionable data. In January, drug maker Biogen announced it was abandoning Aduhelm, a highly contentious Alzheimer’s drug that failed two large trials prior to its heavily criticized approval.

>>> Read full article>>>
Copyright for syndicated content belongs to the linked Source : Ars Technica – https://arstechnica.com/?p=2015321