Wednesday, a panel of federal health advisors voted to recommend clearance for an experimental treatment to treat Lou Gehrig’s illness, a remarkable turnaround for the much-debated prescription that had previously been denied by the same body.
Despite continued questions regarding the validity and dependability of the company’s lone trial, the Food and Drug Administration advisory panel ruled 7-2 that Amylyx Pharma’s results merited approval. The vote is nonbinding, and the FDA will make a final determination before the end of the month.
In spite of a negative review issued by FDA’s own scientists prior to the conference, the majority of external panelists agreed that Amylyx had provided sufficient evidence to demonstrate that the treatment probably helps patients live longer.
Dr. Liana Apostolova of Indiana University’s School of Medicine, who voted for approval, stated, “To deny ALS patients access to a medicine that may work is probably not something I would feel particularly comfortable with.” “At the prior meeting, it was unclear, and it remains uncertain.”
Amylyx also appears to have benefited from an unusual conversation in which a business official agreed, at the FDA’s request, to take the drug from the market if its benefits are not proven by a large, continuing research.
“I’m reasonably confident that if an approval is granted, it can be revoked in the future,” Apostolova said.
Wednesday’s vote ended a rare second meeting to analyze various additional statistical analyses filed by Amylyx in support of the treatment’s ability to delay disease progression and increase life expectancy. In March, the same committee of neurology specialists voted narrowly against the medicine, citing concerns over missing data and implementation flaws in the company’s trial.
The FDA has only approved two treatments for amyotrophic lateral sclerosis (ALS), a condition that destroys nerve cells necessary for basic tasks such as walking, talking, and swallowing.
Patients with ALS and their families have mobilized in support of Amylyx’s medicine, launching an intensive lobbying campaign and recruiting members of Congress to urge FDA clearance.
As a measure of the FDA’s flexibility in examining experimental treatments for terminally ill patients and its capacity to withstand external pressure, the ALS drug review is being keenly monitored.
Dr. Billy Dunn, the FDA’s chief of neurology review, opened the discussion by describing the “concerns and limits” of Amylyx’s findings and highlighting the need for new therapeutic choices.
Dunn stated, “We are acutely aware of the critical need for the discovery of novel ALS medicines.”
A larger Amylyx research being done in the United States and Europe could yield “more definite results” by 2024, according to Dunn.
Dunn said, in a highly unprecedented move, that the government would be more willing to approve the treatment if Amylyx commits to remove the prescription if the continuing 600-patient trial fails to demonstrate a benefit. He then requested that the firm’s co-founders publicly commit to this action, and Amylyx co-CEO Justin Klee stated that the company would voluntarily remove their drug in such a situation.
The FDA has the authority to compel drugmakers to remove their products off the market, though it’s typically quicker if drugmakers do so willingly. In instances when corporations oppose elimination, the regulatory process might drag on for years.
Dr. Caleb Alexander of Johns Hopkins University, one of the two panelists who voted against the medicine, said, “With all due respect, I believe the FDA dramatically understates the complexity and likelihood of their removing the product from the market.”
According to FDA reviewers, Amylyx conducted one modest, mid-stage trial of its medicine that showed some efficacy in reducing the disease, but it was hampered by missing data and other issues.
Tristan Massie, a statistician for the FDA, told panelists, “The ultimate outcome for a single research is borderline and not statistically convincing.”
The Cambridge, Massachusetts, company asserts that post-study follow-up statistics indicate the medicine lengthens life. According to a new company research, patients who maintained taking the medicine lived almost 10 months longer than patients who never received the drug.
These statistics, combined with the medicine’s minor side effects, were touted by pro-drug panelists as evidence that there would be little disadvantage for patients even if the drug did not ultimately reduce ALS.
Dean Follmann, a biostatistician at the National Institutes of Health, stated, “The medicine is not hazardous; it appears to be beneficial; there is no safety signal here.”
More than twenty ALS experts, patients, and family members expressed support for approval to the committee Wednesday morning. Additionally, the FDA has received over 1,200 written comments, the most of which are from ALS patient advocates.
“I’m requesting your approval because I know it’s effective. It is prolonging my life, and I wish the same for others “Greg Canter, diagnosed with ALS in 2018 and a participant in Amylyx’s trial, stated: He attributes his improved lung capacity and slowed functional deterioration to the medication.
The medication sold by Amylyx is a powder that combines two older drugs: a prescription drug for liver diseases and a dietary supplement used in traditional Chinese medicine.
The FDA’s controversial approval of the Alzheimer’s medicine Aduhelm last year, which was assessed by the same government experts and private consultants, hangs over the review.
In this instance, the FDA disregarded the overwhelmingly negative vote of its external advisors, three of whom resigned as a result of the decision. Congress and the federal inspector general are investigating the agency’s approval, which followed questionable meetings with the manufacturer Biogen.