FDA approves controversial new drug designed to slow the progress of ALS

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

The drug, from Massachusetts-based Amylyx Pharmaceuticals, joins just a handful of drugs approved by the agency for the deadly neurodegenerative disease and its symptoms.

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

The FDA’s decision was based on a single Phase 2 clinical study of 137 ALS patients that found people taking Amylyx’s drug, which will be sold under the name Relyvrio, lived about 10 months longer than those who didn’t. The drug was also found to slow hospital admissions.

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

Amylyx said Friday that Relyvrio costs about $12,500 for a 28-day supply, or $158,000 a year without insurance. That’s less than the price of an older ALS drug, edaravone, which costs about $170,000 a year. But Relyvrio’s price is still above the Institute for Clinical and Economic Review’s recommended price of $9,100 to $30,700 per year.

The approval is likely to spark controversy among neurologists treating ALS.

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

Concerns over the study results were raised in March, when the drug was first submitted to an FDA advisory committee. In briefing papers released ahead of 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 calling a second advisory committee just six months later, after Amylyx submitted additional analysis of its research data. At that meeting, the advisory committee reversed course and voted to recommend the drug.

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

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 against the recommendation by an overwhelming majority, due to a lack of evidence that it was effective.

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

However, the decision could pose further challenges for the agency, she added, as it may not give the company much incentive to prove the drug works.

“It also puts the responsibility on payers to decide whether the evidence is enough to support coverage, so in that sense it might just be a kick in the butt,” she said.

Proponents say that while questions remain about the drug’s effectiveness, patients should at least be able to try it.

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

Amylyx is conducting a larger Phase 3 clinical trial, expected to be completed in late 2023 or early 2024.

At the September advisory committee meeting, company representatives agreed to withdraw the drug from the market if the study results showed it to be 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 Amylyx drug is taken. It is taken orally as a powder, not a pill.

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The Valley Voice
The Valley Voicehttp://thevalleyvoice.org
Christopher Brito is a social media producer and trending writer for The Valley Voice, with a focus on sports and stories related to race and culture.

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