ALS drug gets rare second review at high-stakes FDA meeting

WASHINGTON (AP) — A closely watched experimental drug for Lou Gehrig’s disease got an unusual second look from U.S. regulators Wednesday after intense pressure to approve the treatment for sufferers of the deadly disease.

Patients and their families have rallied behind the drug from Amylyx Pharma, launching an aggressive lobbying campaign and recruiting members of Congress to pressure the Food and Drug Administration to grant approval.

The FDA has approved only two treatments for the disease, amyotrophic lateral sclerosis, which destroys nerve cells needed for basic functions such as walking, talking and swallowing. The more effective of the two drugs extends life by several months.

In a rare move, the FDA convened a second meeting of neurology advisers who narrowly voted against the company’s drug in March. The committee was reviewing new statistical analyzes from Amylyx and planned to vote again on whether to recommend approval. The FDA is not required to follow the group’s guidelines.

An internal review by FDA scientists published before the meeting struck a negative tone, concluding that the company’s updated analysis was not “convincing” and provided “no new data.” On the other hand, the FDA’s instructions to the panel emphasized the need for regulatory flexibility when reviewing drugs for fatal diseases.

The FDA’s final decision is expected later this month.

Dr. Billy Dunn, the FDA’s director of neurology inspection, opened the meeting by noting the “concerns and limitations” with the Amylyx data, while stressing the need for new options for patients.

“We are particularly sensitive to the urgent need to develop new treatments for ALS,” Dunn said. “We have not made any final decisions regarding the approval of this application.”

Dunn also noted that a larger study being conducted by Amylyx could provide “more definitive results” on the drug’s effectiveness by 2024.

The ALS drug review is being watched closely as an indicator of the FDA’s flexibility in reviewing experimental drugs for terminal patients and its ability to withstand outside pressure.

“We’re here because there’s a lot of pressure,” said Diana Zuckerman of the nonprofit National Center for Health Research, which analyzes data and conducts medical research. “The FDA goes the extra mile by saying you can have another meeting, but the company responded by giving them new data.”

Amylyx conducted a small, mid-stage trial of its drug that showed some benefit in slowing the disease, but was plagued by missing data and other problems, according to FDA reviewers.

The Cambridge, Mass.-based company says follow-up data collected after the study showed the drug extended lifespan. Patients who continued to take the drug survived about 10 months longer than patients who never took the drug, according to a new analysis by the company.

But the FDA scientists said in their review that the new approach “suffers from the same interpretive challenges” as the original study.

On Wednesday, the FDA will again hear from patients and advocacy groups like I AM ALS, which has been lobbying for more than two years to make the drug available.

“We have a drug that the entire ALS community is behind. Patients, clinicians, researchers all support this because of what we’ve seen in data from the trial,” said I AM ALS co-founder Brian Wallach, speaking through an interpreter. “That’s not the case with every drug.”

Amylyx’s drug comes as a powder that combines two older drugs: a prescription drug for liver disorders and a dietary supplement used in traditional Chinese medicine. Wallach and some other ALS patients already take the dietary supplement.

The review hinges on the FDA’s controversial approval of the Alzheimer’s drug Aduhelm last year, which was reviewed by agency scientists and outside consultants.

In that case, the FDA ignored the overwhelmingly negative vote of its outside counsel, three of whom resigned over the decision. The FDA’s approval — which followed informal meetings with drug company Biogen — is under investigation by congressional committees and federal inspectors.


Follow Matthew Perrone on Twitter: @AP_FDAwriter


The Associated Press Health and Science Section is supported by the Howard Hughes Medical Institute Science Education Division. AP is solely responsible for all content.

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