ALS drug pulled from market

Four weeks after reporting its controversial and costly drug for amyotrophic lateral sclerosis failed to provide any benefit to patients in a large clinical trial, Cambridge biotech Amylyx Pharmaceuticals said Thursday it was pulling the medicine from the market in the US and Canada. The firm also said it plans to lay off 70 percent of its workforce to preserve its cash into 2026. Amylyx had 384 full-time employees as of December 31. The layoffs will reduce the workforce to a little more than 100, said a company spokesperson. The drug, which is called Relyvrio in the United States and Albrioza in Canada, “will no longer be available for new patients as of today,” Amylyx said in a news release. Patients who are currently on the medicine and want to continue taking it will be able to get it for free. “While this is a difficult moment for the ALS community, we reached this path forward in partnership with the stakeholders who will be impacted and in line with our steadfast commitment to people living with ALS and other neurodegenerative diseases,” Joshua Cohen and Justin Klee, cofounders and cochief executives of Amylyx, said in a statement. Relyvrio cost about $163,000 a year per patient. It generated nearly $381 million in revenue last year for the 11-year-old company. While the decision to withdraw it from the market was commended by experts, it is a major blow to ALS patients, their families, and doctors, given the dearth of effective treatments for the fatal disease. It also caps a years-long drama during which the prospects of Amylyx’s drug fluctuated wildly, until the company made a major announcement last month. In a stunning reversal on March 8, Amylyx released the final results of a 48-week, late-stage confirmatory study involving 664 people with ALS at more than 70 trial sites in the United States and Europe. Amylyx’s drug failed to outperform a placebo on an ALS functional rating scale that measured patients’ ability to perform daily living tasks, breathe, walk, speak, and swallow. Cohen and Klee said then that they were “surprised and deeply disappointed” and would consider withdrawing the drug from the market.

The drug was approved in September 2022, even though an advisory panel to the Food and Drug Administration initially recommended the agency reject it, citing weak scientific evidence of its benefits. That panel later reversed itself amid wrenching pleas from patients and families affected by the paralyzing and fatal neurological condition, and after Amylyx presented more clinical data that it said showed the medicine extended patients’ lives — JONATHAN SALTZMAN