The Food and Drug Administration on Thursday approved a controversial new drug designed to slow the progression 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, joins just a handful of agency-approved drugs for the deadly neurodegenerative disease and its symptoms.
The disease, also called amyotrophic lateral sclerosis, or ALS, affects nerve cells needed for activities like walking, talking, 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 trial of 137 ALS patients that found people who took Amylyx’s drug, which will be sold under the name Relyvrio, lived about 10 months longer than the others. The drug also appears to delay hospitalizations.
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 taurursodiol, an over-the-counter supplement. 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 around $170,000 a year. But Relyvrio’s price is still higher than the Institute for Clinical and Economic Review’s recommended price of between $9,100 and $30,700 per year.
The endorsement is likely to engender some disagreement among neurologists who treat ALS.
Typically, the FDA requires at least two well-controlled clinical trials to demonstrate that a drug is effective or a single “statistically very convincing” trial, said Holly Fernandez Lynch, assistant professor of medical ethics at the University of Pennsylvania. Amylyx’s trial, she said, failed to meet agency standards.
Concerns about the trial results were raised in March, when the drug was first presented to an FDA advisory panel. In background papers released ahead of the meeting, agency scientists questioned the persuasiveness of the Amylyx trial. The committee narrowly voted against the recommendation for approval.
The FDA, however, took the unusual step of convening a second advisory committee six months later, after Amylyx submitted additional analysis of its trial data. During that meeting, the advisory committee reversed course, voting to recommend the drug.
The positive vote came despite another lackluster review from FDA scientists, as well as concerns from several advisory committee members about whether clinical trial data provided by Amylyx showed clear evidence that the drug slows the progression of the disease. sickness.
The agency’s consideration of the drug was compared to that of Biogen’s Alzheimer’s drug, Aduhelm. This drug received full FDA approval last year, even though the advisory committee voted overwhelmingly against its recommendation, citing a lack of evidence of its effectiveness.
Amylyx’s drug approval suggests the FDA is willing to show the most flexibility for life-threatening conditions for which there is an “unmet treatment need,” Lynch said.
However, the decision could pose other challenges for the agency, she added, as it may not prompt the company to prove that the drug works.
“It’s also up to the payers to decide whether the evidence is sufficient to support coverage, so in that sense it may just kick in,” she said.
Proponents say that while questions remain about the drug’s effectiveness, patients should be allowed to at least try it.
“We need new treatments as soon as possible if we are to turn ALS into a viable disease and eventually cure it,” Larry Falivena, a member of the ALS Association, an advocacy group, said in an email.
Amylyx is currently conducting a larger phase 3 clinical trial, which it expects to complete in late 2023 or early 2024.
At the September advisory committee meeting, company representatives agreed to pull the drug from the market if the results of those trials showed it was not effective. The drug has already received conditional approval in Canada.
CORRECTION (September 29, 2022, 9:34 p.m. ET): An earlier version of this article misrepresented how the drug Amylyx is taken. It is taken orally in powder form, not in pill form.
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