Amylyx Pharmaceuticals, the maker of a drug for Lou Gehrig’s disease, announced that it will be pulling the medicine from the market after it failed in a large study. The drug, Relyvrio, did not show any benefit for patients with the deadly neurological condition, leading the company to make the difficult decision to halt sales and marketing in the U.S. and Canada. Patients already taking the therapy will have the option to continue receiving it for free through a special program. The FDA approved Relyvrio in September 2022 after a long advocacy campaign by patients with amyotrophic lateral sclerosis, or ALS.
The failure of Relyvrio is a devastating blow to patients and advocates who have been pushing for more experimental therapies for ALS. With the withdrawal of Relyvrio, there are now only three ALS medicines available to U.S. patients, and only one of them has been shown to extend survival by several months. Amylyx also announced that it will be laying off 70% of its more than 350 employees as part of a major restructuring effort. However, the company plans to continue studying Relyvrio and another experimental drug for several rare diseases, including Wolfram syndrome, which causes childhood diabetes and blindness.
After a clinical trial with 600 patients showed no improvements in survival or other health measures, Amylyx considered pulling its drug from the market. The company’s voluntary action to remove Relyvrio resolves a potential dilemma for the FDA, as the agency would not have had a clear path to quickly force the drug off the market if the company had refused. The FDA had granted full approval to Relyvrio based on data from a small, mid-stage study, which had been criticized by some internal scientists. Typically, the FDA requires two large, late-stage studies that demonstrate clear benefits before granting approval, but in this case, regulatory flexibility was deemed appropriate due to the serious nature of ALS and the significant unmet need.
Relyvrio is a powder that combines two older drugs, one for liver disorders and the other a dietary supplement associated with traditional Chinese medicine. The drug was priced at $158,000 for a year’s supply, which led to disappointing sales, with some patients discontinuing the medicine after only a few months. ALS is a disease that gradually destroys nerve cells, impacting a person’s ability to walk, talk, speak, and breathe, typically resulting in death within three to five years of diagnosis. Despite the setback with Relyvrio, the company’s shares climbed more than 7.5% in trading following the announcement of the drug’s withdrawal from the market.
