FDA Approves Controversial New Drug Designed to Slow ALS Progress

The Food and Drug Administration (FDA) on Thursday approved a controversial new drug designed to slow the progress of Lou Gehrig’s disease, a victory for patients and advocates despite limited evidence that the drug is effective.

The drug, from Amylyx Pharmaceuticals of Massachusetts, binds itself a handful of drugs approved by the agency for fatal neurodegenerative disease and its symptoms.

The disease, also called amyotrophic lateral sclerosis, or ALS, affects the nerve cells needed for activities like walking, talking, and eating. There is no known cure, and most people live for only two to five years after diagnosis. according to the ALS Association.

The FDA’s decision was based on a phase 2 clinical trial of 137 ALS patients that found people who took Amylyx’s drug, which will be sold under the brand name. relief, lived about 10 more months than those who did not. The drug also seemed to delay hospitalizations.

The medication, which comes in powder form, is a combination of two existing products: sodium phenylbutyrateprescribed to treat a metabolic disorder, and taurursodiol, an over-the-counter supplement It is used to help prevent liver disease.

Amylyx said Friday that Relyvrio will cost about $12,500 for a 28-day supply, or $158,000 per year before insurance. That’s below the price of an older ALS drug, edaravone, which costs about $170,000 a year. But the price of Relyvrio is still above the Institute for Clinical and Economic Review. recommended price between $9,100 to $30,700 per year.

The approval is likely to cause some disagreement among neurologists who treat ALS.

Generally, the FDA requires at least two well-controlled clinical trials to show a drug is effective or a single trial that is “highly statistically persuasive,” said Holly Fernandez Lynch, an assistant professor of medical ethics at the University of Pennsylvania. Amylyx’s trial, he said, did not meet agency standards.

Concerns about the trial results were raised in March, when the drug was first brought before an FDA advisory committee. In informative documents Released before the meeting, agency scientists questioned the persuasiveness of the Amylyx trial. The committee narrowly voted against recommending approval.

However, the FDA took the unusual step of convening a second advisory committee just six months later, after Amylyx submitted additional analysis of its trial data. During that meeting, the advisory committee changed course, voting to recommend the drug.

The favorable vote came despite another poor review by FDA scientists, as well as concerns from several advisory committee members about whether the clinical trial data provided by Amylyx showed clear evidence that the drug slows progression. of the illness.

The agency’s consideration of the drug has been compared to that of Biogen’s Alzheimer’s drug Aduhelm. That drug received full FDA approval last year, though the advisory committee voted overwhelmingly against his recommendationciting a lack of evidence that it was effective.

The approval of the Amylyx drug suggests that the FDA is willing to show maximum flexibility for life-threatening diseases for which there is an “unmet need for treatment,” Lynch said.

However, the decision could pose more challenges for the agency, he added, because it may not give the company much incentive to prove the drug works.

“It also puts the onus on payers to decide if the evidence is sufficient to support coverage, so in that sense you can just kick the boat,” he said.

Advocates say that while questions remain about the drug’s efficacy, patients should be allowed to at least try it.

“We need new treatments as quickly as possible if we are to make ALS into a tolerable disease and eventually cure it,” Larry Falivena, a member of The ALS Association, an advocacy group, said in an email.

Amylyx is running a larger phase 3 clinical trial, which it hopes to complete in late 2023 or early 2024.

During the September advisory committee meeting, company representatives agreed to withdraw the drug from the market if trial results showed it was ineffective. The drug has already received conditional approval in Canada.

CORRECTION (September 29, 2022, 9:34 pm ET): An earlier version of this article incorrectly stated how the drug Amylyx is taken. It is taken orally as a powder, not a pill.

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