ALS drug to be withdrawn from U.S. market after study shows no benefit to patients

WASHINGTON (AP) - The maker of a drug for Lou Gehrig's disease that recently failed in a large study said Thursday that it is pulling it from the market, acknowledging that it doesn't help people with the deadly neurological disorder.

Amylyx Pharmaceuticals, Inc. announced that it would voluntarily discontinue sales and marketing of the drug in the United States and Canada, and that new patients would no longer be able to receive the treatment.

"While this is a difficult time for the ALS community, we have worked with the stakeholders who will be impacted to achieve this path forward based on our strong commitment to ALS patients," said Kougoun in a statement. Patients already taking the treatment who wish to continue can add a free program.

The U.S. Food and Drug Administration (FDA) approved the controversial drug Relyvrio in September 2022, after years of campaigning by people with amyotrophic lateral sclerosis (ALS).

The drug's failure has been a major disappointment to patients and advocates, who have been urging the FDA and other federal agencies to fund the approval of more proven treatments for the deadly muscular dystrophy.

Relyvrio's withdrawal leaves U.S. patients with access to only three ALS drugs, only one of which has been shown to extend survival by several months.

Amylyx, an Artech company based in Cambridge, Massachusetts, said in March that it was considering withdrawing its drug after conducting clinical trials on 600 patients because it had not shown any improvement in survival or other health indicators, such as muscle strength or walking ability.

The company's voluntary action solved a major problem the FDA might have faced. If the company had refused to take the drug off the market, there would have been no clear way for the agency's regulators to quickly force the drug off the market. This is because the FDA fully approved the drug, even though the company's efficacy data were only preliminary.

The 2022 approval was based largely on the results of a small interim study that was criticized by some scientists within the agency. Typically, the agency requires two large post-study studies to show significant benefits before approval. But whereas FDA officials explained that "given the serious and life-threatening nature of ALS and the large unmet need," it was appropriate to "monitor Koon's activity" during the trial of Relyvrio.

In recent years, the U.S. Food and Drug Administration (FDA) has approved a series of drugs for the treatment of fatal degenerative diseases, but this drug is one of them, despite the fact that the evidence of the effectiveness of these drugs is questionable.

SLE gradually destroys the nerve cells and connections needed to walk, talk, speak and breathe. Most patients die within three to five years of diagnosis.

Relyvrio is a powder which combines two older medicines: a prescription medicine for liver diseases and a dietary supplement related to traditional Chinese medicine.

Amylyx was criticized for pricing the drug at $158,000 a year. Sales were disappointing, with some patients stopping taking the drug after only a few months.

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The Associated Press Health and Science Division is supported by the Science and Education Media Unit of the Howard Hughes Medical Institute. The Associated Press is solely responsible for all content.