FDA approves ALS drug funded by Ice Bucket Challenge


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. The oral medication works as a stand-alone therapy or when added to other treatments, according to the company, and has been shown to slow the progression of the disease.

“This approval provides another important treatment option for ALS, a life-threatening disease that currently has no cure,” said Dr. Billy Dunn, director of the Office of Neuroscience at the FDA’s Center for Drug Evaluation and Research.

Patients and some advocacy groups urged the FDA to approve the drug, which has limited treatments for ALS, and the agency granted it priority review in December.

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

“ALS is a terrible disease: rapidly fatal and truly debilitating from initial symptoms to death. The FDA has approved a couple of treatments, but they are minimally effective and certainly not a cure. And so there is a huge unmet need in this area of ​​the disease,” the FDA said. admitted,” said Holly Fernandez Lynch, assistant professor of medical ethics and health policy at the University of Pennsylvania.

Before the FDA’s decision, Lynch told CNN he would be “surprised” if the drug was not approved because the FDA’s Central and Peripheral Nervous System Drugs Advisory Committee changed its mind on the drug at a meeting this month. 7-2 in favor of approval.

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

The trial also showed that the drug was generally well tolerated, but there was a higher frequency of gastrointestinal events in the group receiving the drug. Amylyx continues to study its safety and efficacy in a Phase 3 trial.

In March, the Central and Peripheral Nervous System Drugs Advisory Committee voted 6-4 that not a single phase 2 trial concluded that the drug is effective in treating ALS.

“In terms of concluding that it’s effective, we were asked persuasively and robustly to find substantial evidence and I think this trial doesn’t quite deliver,” Dr. Kenneth Fischbeck, one of the committee members and a researcher at the National Institutes of Health, said at the March meeting. He added that Fischbeck has treated ALS patients.

A key difference between the March and September FDA advisory committee meetings is that at the later meeting, Amylyx indicated that if the drug was approved but the results of its Phase 3 trial did not confirm the drug’s benefits, the company would consider taking the drug off the market. , Lynch said. He added, however, that the company did not say exactly what it would consider a failure.

“So in the vote, the members of the advisory board changed, and most of them said, ‘Yes, we now believe this product should be approved.’ And when asked why they changed their minds, some said, ‘Well, the company said they were going to withdraw. ‘” he said. “And the patients’ testimonials also convinced them that they want to try this medicine a lot.”

But the FDA’s approval was generally based on data from Phase 2 trials, which Lynch said could send a message to other pharmaceutical companies that they don’t need strong Phase 3 trial data to get their products to market.

Lynch said that while he understands why people with ALS want access to this promising drug, he worries that the message could open the door wider to the approval of unproven drugs. The FDA can later withdraw those products if necessary, he said, but doing so without the company’s voluntary agreement is “a huge pain” and often requires a very long process.

As for Relyvrio, some ALS advocacy groups – including the ALS Association – have been pushing for its approval for several months. After an FDA advisory board meeting in March, when the board initially voted against the drug, Calaneet Balas, president and CEO of the ALS Association, said in a statement that “the FDA has to make a choice: whether or not to approve a drug that has been proven safe today.” that it will help people with ALS today, or that it will delay approval and require more evidence as more people with ALS die.”

“We cannot let perfection get in the way of real progress in turning ALS from a life-threatening disease. The FDA’s ALS Guidance recognizes that people with ALS are willing to accept greater risk for some benefit,” Balas said. “People with ALS and their families deserve better, and the FDA has the tools to urgently accomplish that.”