ALS Drug Gains FDA Approval Despite Uncertainty About Its Effectiveness |  CNN

ALS Drug Gains FDA Approval Despite Uncertainty About Its Effectiveness | CNN


A new treatment for amyotrophic lateral sclerosis, or ALS, has been approved by the US Food and Drug Administration.

The FDA announced Thursday the approval of Relyvrio, developed by Amylyx Pharmaceuticals. According to the company, the oral drug can be taken as a stand-alone therapy or alongside other treatments, and has been shown to slow disease progression.

But there’s still some uncertainty about the drug’s effectiveness: Amylyx’s application for approval is based on data from a small phase 2 trial, and the FDA’s own advisory committee initially voted that spring that the data did not show the drug was effective, before changing its advice this month.

“There are limitations to these results that result in a degree of residual uncertainty about the evidence of efficacy that exceeds what might generally remain after a conclusion that substantial evidence of efficacy has been demonstrated,” states a memorandum from FDA summary on approval. But “given the serious and life-threatening nature of ALS and the significant unmet need, this level of uncertainty is acceptable in this case.”

The approval is the first in the United States for Amylyx, CEOs Josh Cohen and Justin Klee said in a statement, and “is an exciting step” for the ALS community.

“Amylyx’s goal is for everyone who is eligible for Relyvrio to have access to it as quickly and efficiently as possible, because we know people with ALS and their families don’t have time to wait,” they wrote. declared. “While Amylyx works to launch Relyvrio, healthcare professionals will be able to write prescriptions for Relyvrio immediately by enrolling their patients in our comprehensive support program that we are implementing.”

Patients and some advocacy groups had urged the FDA to approve the drug because available treatments for ALS are limited, and the agency granted priority review in December.

ALS, also known as Lou Gehrig’s disease, affects up to 30,000 people in the United States. It is a neurodegenerative disease that weakens the muscles, eventually affecting the ability to speak, swallow, move and breathe.

“ALS is a horrible disease: quickly fatal and very debilitating during the period from the first symptoms to death. The FDA has approved a few treatments, but they are not very effective and certainly do not cure. And so, there’s a vast unmet need in this area of ​​disease, which the FDA has recognized,” said Holly Fernandez Lynch, assistant professor of medical ethics and health policy at the University of Pennsylvania.

Prior to the FDA’s decision, Lynch told CNN she would be “shocked” if the drug was not approved because the FDA’s Central and Peripheral Nervous System Drug Advisory Committee changed its opinion on the drug. drug at a meeting this month, voting 7-2 in favor of approval.

In November, Amylyx submitted a drug application to the FDA for the drug, then called AMX0035, as an oral treatment for ALS, seeking approval based on a Phase 2 trial that included 137 people with ALS who received either the drug or a placebo for 24 weeks. . The study was partially funded by a grant from the ALS Ice Bucket Challenge, the viral social media campaign launched in 2014 involving people throwing buckets of ice water at themselves to raise awareness and funds around ALS.

The trial also showed that the drug was generally well tolerated, but there was a greater frequency of gastrointestinal events in the group receiving the drug. Amylyx is currently studying its safety and efficacy in a phase 3 trial.

In March, the Peripheral and Central Nervous System Drugs Advisory Committee voted 6 to 4 that a single phase 2 trial failed to conclude that the drug is effective in treating ALS.

“In terms of reaching the conclusion that it is effective, we were asked to search for substantial evidence persuasively and robustly and I think this trial does not quite meet that bar,” Dr Kenneth said. Fischbeck, one of the committee members and a National Institutes of Health investigator, said at the March meeting. Fischbeck added that he cared for ALS patients.

A key difference between the March and September meetings of the FDA advisory committee is that at the subsequent meeting, Amylyx indicated that if the drug was approved but the results of its Phase 3 trial did not confirm the benefits of drug, the company would consider withdrawing the drug from the market. , said Lynch. She added, however, that the company did not specify what it would consider a failure.

“So during the vote, the members of the advisory committee changed, and most of them said, ‘Yes, we are now convinced that this product should be approved.’ And when asked why they changed their minds, some of them said, ‘Well, the company said they would pull out,’ she said. been convinced by patient testimonials that they really want to try this drug.”

But overall, the FDA approval was based on phase 2 trial data, which Lynch says may send a message to other drug companies that they don’t need phase 2 trial data. solid phase 3 to bring products to market.

Lynch said that while she understands why people with ALS want access to this promising drug, she fears that such a message could open the door wider to the approval of drugs whose effectiveness has not yet been tested. not been proven. The FDA could later withdraw these products if necessary, she said, but doing so without the company’s voluntary agreement is “a huge pain” and often requires a very lengthy process.

As for Relyvrio, some ALS advocacy groups – including the ALS Association – have been calling for its approval for several months. After the FDA’s advisory committee meeting in March, when the panel initially voted against the drug, Calaneet Balas, president and CEO of the ALS Association, said in a statement that “the FDA has a choice to do – whether it will approve a drug that has been proven safe that will help people living with ALS today, or whether it will delay approval and require more evidence as more people with ALS die.

“We can’t let perfection stand in the way of real progress towards transforming ALS from a fatal disease to a livable disease. The FDA’s own ALS guidelines recognize that people with ALS are willing to accept greater risk for the possibility of some benefit,” Balas said. “People with ALS and their loved ones deserve better, and the FDA has the tools to make that happen urgently.”

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