A federal panel of independent medical experts ruled Wednesday that an experimental treatment for amyotrophic lateral sclerosis, widely known as A.L.S., is ineffective in treating the debilitating and lethal neurological illness.

According to the FDA’s 10-member advisory group, AMX0035, a two-drug combo made by two university students, was unable to show it could benefit patients by delaying the progress of A.L.S., a cruel disease that destroys people’s ability to move, speak, eat, and ultimately breathe.

According to the Food and Drug Administration, a nonbinding vote on the medication AMX0035 endorsed the F.D.A.’s own findings by a vote of 6 to 4. The FDA will make a decision on whether or not to approve the medicine in the coming months.

Given the lack of treatments for A.L.S., commonly known as Lou Gehrig’s disease, which often results in death within two to five years, patient advocacy groups have launched a passionate fight for approval.

“There’s no question of the burdensome nature of the disease and a huge unmet need for safe and effective treatment,” says panellist Dr. Kenneth Fischbeck, an investigator with the National Institute of Neurological Disorders and Stroke. “On the other hand,” he said, “we were asked to look for substantial evidence with persuasiveness and robustness. And I think this one trial doesn’t quite meet that bar.”

Mark Weston, a patient representative on the panel who suffers from A.L.S., voted that the treatment was beneficial. “This was a very difficult decision for me and I could have gone either way,” said Dr. Avindra Nath, clinical director of the National Institute of Neurological Disorders and Stroke. “But after weighing all the factors and facts presented I hedged over to the yes side.”

The panelists were all struck by the passionate testimony of persons living with A.L.S.

Vance Burghard, a clinical trial participant, stated that after being diagnosed in 2017, “I needed assistance to pull up my pants, had to have my food cut for me,” and “needed to use a wheelchair on occasion.” “AMX0035 is a lifesaving and life-changing drug for me,” he added, credited it with allowing him to travel with his wife, walking “many miles in Europe, at the Great Wall of China, and descending the stairs at the Potala Palace in Tibet”.

Becky Mourey, 58, was one of several patients whose speech had deteriorated to the point where family members had to read portions of their sentences to them. “Those inflatable punching bags many of us had as kids,” she said, comparing A.L.S. to “those inflatable punching bags many of us had as kids — one stroke, it would bounce back up just to be hit again and again and again.”

“I implore you with literal life and death urgency to recommend approval of AMX0035,” Ms. Mourey said, adding that she had been taking the two medications that make up the therapy on her own.

The Food and Drug Administration usually requires two convincing clinical trials, but in cases of severe disease with limited treatments available, it can consider evidence from one clinical study plus extra supporting data. The results comparing AMX0035 to a placebo came from a Phase 2 trial, which was smaller than the preferred Phase 3 research, and an open-label extension study that followed some patients after the trial finished while they were knowingly taking the medication.

Until this summer, the Food and Drug Administration had advised Amylyx Pharmaceuticals not to file for approval until the drug’s Phase 3 trial was completed, with results expected in 2024. Officials began recommending in July that Amylyx submit an application for approval based on current data. Following the acceptance of Aduhelm, there was a lot of pressure from A.L.S. advocacy groups.

Every year, roughly 6,000 people worldwide are diagnosed with A.L.S. Only two A.L.S. treatments are now approved: riluzole, which can extend survival by several months, and edaravone, which can decrease growth by around 33%.

In the Phase 2 trial, two-thirds of the 137 individuals were given AMX0035, a bitter-tasting powder mixed with water to be sipped or eaten twice daily through a feeding tube. The others were given a placebo.

The major goal was to slow the decline of 12 physical abilities, including walking, speaking, swallowing, dressing, handwriting, and breathing, on a 48-point A.L.S. scale. Over the course of 24 weeks, those who received a placebo dropped 2.32 points more than those who received AMX0035, resulting in a 25% slower decline for those who received the medication.

The open-label extension study included 90 patients, including 34 from the placebo group, who started taking AMX0035 seven months after those who had gotten it from the start. According to Amylyx, those who received the treatment for the longest had a median of 4.8 months longer before being hospitalized, being put on a ventilator, or dying.

“This is the first time that we have seen a benefit in both function and survival in an A.L.S. clinical trial,” adds Dr. Sabrina Paganoni, the principal investigator of the clinical trial.

“If access is delayed, the patients in my clinic today may never receive the time and function that they could have had,” she added.

The research, on the other hand, was found to have numerous flaws by FDA reviewers.

Dr. Emily Freilich, a member of the FDA’s team, said on Wednesday that the Phase 2 trial results indicating that AMX0035 slowed functional decline were “not highly persuasive,” and that the trial’s secondary measures — such as muscle strength, respiratory ability, and whether patients were hospitalized — were “not generally supportive” of benefit.

Officials with the Food and Drug Administration said the evidence, even from the extension trial, does not show that patients who receive the therapy live longer. Patients who received the placebo and never switched to AMX0035 lived for a median of 1,295 days, according to Dr. Freilich, while those who took AMX0035 for more than 96 weeks lived for a median of 1,237 days.

“Therefore, we need to ask ourselves if the noted survival benefit is by chance alone or due to underlying disease heterogeneity, rather than an effect of the drug,” she said.

Concerns raised by the FDA included how deaths were accounted for; whether patients could tell they were on AMX0035 from its bitter taste and gastrointestinal side effects; the fact that more patients receiving the treatment were taking one of the other A.L.S. drugs; and the fact that the trial’s data analysis method was not what the agency urged Amylyx to use.

The Food and Drug Administration agreed with Amylyx that AMX0035 did not pose any substantial safety risks.

As an internist and epidemiologist at the Johns Hopkins Bloomberg School of Public Health, Dr. Alexander disagrees with the idea of approving drugs that have little or no effect, just because they look safe and desperate patients would be willing to try them.

“If you did,” he said, “we would have dozens of therapies on the market that might not actually be therapeutic.”

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