(CNN) — The United States Food and Drug Administration (FDA) has approved a new treatment for amyotrophic lateral sclerosis, also known by its acronym, ALS.
The FDA gave the green light to Relyvrio, an oral drug developed by Amylyx Pharmaceuticals, which works as a stand-alone therapy or can be complementary to other treatments. According to the company, it has been shown to slow the progression of the disease.
However, there is still some uncertainty surrounding the drug’s efficacy: Amylyx’s application for approval is based on data from a small phase 2 trial, and the FDA’s own advisory committee had initially voted against approval earlier. to change your mind this month.
“This approval provides another important treatment option for ALS, a life-threatening disease for which there is currently no cure,” said Dr. Billy Dunn, director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research, it’s a statement.
Patients and some activist groups have urged the FDA to approve the drug, as available treatments are limited. Because of that, the agency had granted a priority review in December.
Doubts remain about its effectiveness
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, affects about 30,000 people in the United States. It is a neurodegenerative disease that causes muscles to weaken, ultimately affecting the ability to speak, swallow, move, and breathe.
“ALS is a horrible disease: rapidly fatal and really debilitating during the period from initial symptoms to death. The FDA has approved a couple of treatments, but they are minimally effective and certainly not a cure. So there is a huge unmet need in this disease area, 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 he would be “surprised” if the drug was not approved, because the FDA’s Advisory Committee on Drugs for the Central and Peripheral Nervous System changed its opinion on the drug in a meeting this month, voting 7-2 in favor of approval.
In November, Amylyx submitted an application to the FDA for the drug, then named AMX0035, as an oral treatment for ALS, seeking approval based on a phase 2 trial that included 137 people with ALS who received the drug or a placebo. for 24 weeks. The study was funded in part by a grant from the ALS Ice Bucket Challenge, the viral social media campaign that began in 2014 in which people dropped buckets of ice water on themselves to raise awareness and funds around ALS.
The trial also showed that the drug was generally well tolerated, although there was a higher frequency of gastrointestinal events among those who received the drug. Amylyx is now continuing to study its safety and efficacy in a phase 3 trial.
In March, the FDA’s Central and Peripheral Nervous System Drugs Advisory Committee had voted 6-4, arguing that a single Phase 2 trial was inconclusive about the drug’s effectiveness in treating ALS.
“In terms of concluding that it’s effective, we were asked to persuasively and robustly seek substantial evidence, and I think this trial falls short of that standard,” said committee member and investigator Dr. Kenneth Fischbeck. of the National Institutes of Health, at the March meeting. Fischbeck added that he has treated ALS patients.
A key difference between the March and September FDA advisory committee meetings is that, at the latter, Amylyx indicated that if the drug was approved but the Phase 3 trial results did not confirm its benefits, the company would consider withdrawing it. of the market, Lynch added. However, he added that the company did not specifically say what it would consider a failure.
“So, on the vote, the advisory committee members changed their minds, with the majority saying, ‘Yes, we’re now convinced this product should be approved.’ And when asked why they changed their minds, some said, ‘Well , the company said they would pull out,'” he said. “And they were also convinced by patient testimonials that they really wanted to try this drug.
Overall, though, the FDA approval was based on Phase 2 trial data, which, Lynch said, may send a message to other drug companies that they don’t need strong Phase 3 trial data to market their drugs. products.
From a deadly disease to a livable one
Lynch said that while he understands why people with ALS want access to this promising drug, he is concerned that this could open the door more broadly to the approval of drugs that have not been shown to work. The FDA could later withdraw those products if necessary, she said, but doing so without the company’s voluntary agreement is “a huge pain” and often requires a lengthy process.
As for the Relyvrio, some ALS activists, including the ALS Association, have been calling for its approval for several months. After the FDA 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 decision to make: whether to approve a drug that has been shown to be safe and will help people living with ALS today, or will delay approval and require more evidence as more people with ALS die.”
“We cannot let perfection stand in the way of real progress in turning ALS from a deadly disease to a livable one. The FDA’s own ALS guidance recognizes that people with this disease are willing to accept risks for the possibility of benefit,” Balas said. “People with ALS and their loved ones deserve better, and the FDA has the tools to do it urgently.”