FDA Panel Backs Much Debated ALS Drug

An experimental drug to treat Lou Gehrig’s disease has been recommended for approval by a panel of federal health advisers, a remarkable turnaround for the hotly contested drug that was earlier this year rejected by the same group. In spite of hours of discussion about the validity and dependability of the company’s single study, the Food and Drug Administration advisers decided by a 7-2 vote that the data from Amylyx Pharma warranted approval.

Although the FDA is not compelled to heed their recommendations, their positive assessment suggests that an approval is probably coming this month. Only two treatments for ALS, or amyotrophic lateral sclerosis, which kills nerve cells essential for basic bodily activities including walking, talking, and swallowing, have received FDA approval.

ALS sufferers and their families have backed Amylyx’s medication, mounting a vigorous lobbying effort and engaging members of Congress to pressure the FDA to approve it. The majority of the outside panellists agreed that Amylyx had provided sufficient evidence to suggest that the medication is extending patients’ lives, despite a critical assessment by FDA internal scientists that was released in advance of the meeting.

Due to worries about missing data and other problems in the company’s study, the same group of neurology experts narrowly decided to vote against the drug in March. To deprive ALS patients of a drug that might work, it’s probably not something I would feel terribly comfortable with, said Dr. Liana Apostolova of Indiana University’s School of Medicine, who voted for approval.

It wasn’t quite obvious at the prior meeting, and it still raises some questions. Additionally, Amylyx seemed to gain from an unusual conversation in which a business executive agreed, at the FDA’s request, to recall the medication from the market if its advantages aren’t supported by a sizable, ongoing study.

I’m reasonably confident that if an approval is given, it may be revoked later, Apostolova said. The vote on Wednesday put an end to a rare second meeting to go over many fresh statistical studies Amylyx had provided in favour of the treatment’s advantages in slowing disease and lengthening life.

The FDA’s flexibility in evaluating novel treatments for terminally sick patients and its resilience to outside pressure are being widely scrutinised in the ALS drug review. The discussion began with Dr. Billy Dunn, head of the FDA’s neurology evaluation, outlining the issues and limits with Amylyx’s findings and highlighting the demand for alternative therapeutic choices.

We are acutely aware of the pressing need for the creation of novel ALS medicines, according to Dunn. Dunn added that by 2024, more conclusive findings from a larger Amylyx study being carried out in the US and Europe might be available.

Dunn made the highly unusual suggestion that the agency might be more willing to approve the drug if Amylyx committed to stopping production of its drug if the ongoing 600-patient trial failed to demonstrate a benefit.

The co-CEO of Amylyx, Justin Klee, responded that the company would voluntarily withdraw its drug in that situation after he called on the company’s founders to publicly commit to that action. The FDA has the authority to order businesses to remove medications from the market, but voluntary action is usually faster. When businesses fight against being removed, the regulatory process may take years.

I think the FDA with all due respect significantly understates the complexity and likelihood of their pulling the product from the market, said Dr. Caleb Alexander of Johns Hopkins University, one of the two panellists who voted against the drug. FDA reviewers stated that although Amylyx conducted one small, mid-stage trial of its drug that demonstrated some benefit in slowing the disease, it was plagued by missing data and other issues.

Tristan Massie, an FDA statistician, informed the panel that the results of a single study were on the borderline and not very statistically convincing. The Cambridge, Massachusetts, company says follow-up data acquired after the study concluded revealed the medicine extended life.

According to a recent company analysis, patients who continued taking the medication lived about 10 months longer than those who discontinued it. The data, along with the medication’s mild side effects, were cited by panellists who supported the drug, implying that even if it doesn’t ultimately slow ALS progression, there would be little harm to patients.

According to Dean Follmann, a biostatistician with the National Institutes of Health, the medication is not harmful and appears to have a benefit. There are no safety concerns at this time.

Earlier Wednesday, more than 20 ALS researchers, patients and family members told the advisers they supported clearance. Additionally, the agency has received over 1,200 written comments, many of which came from ALS patient advocates.

I’m requesting your approval because I’m confident it will work. Greg Canter, who was given an ALS diagnosis in 2018 and took part in Amylyx’s study, claimed it was extending his life and he wanted the same for other people. He attributes the medication’s improvement in lung function and reversal of his functional deterioration. Amylyx’s treatment comes as a powder that combines two older drugs: one prescription medication for liver diseases and a dietary supplement used in traditional Chinese medicine.

The FDA’s contentious approval of the Alzheimer’s medication Aduhelm last year, which was examined by the same agency experts and outside advisers, hangs over the assessment. In that instance, the FDA disregarded the unanimously negative vote cast by three of its outside experts, who left in protest of the choice. The agency’s permission, which came after erroneous discussions with the pharmaceutical company Biogen, is being looked into by Congress and federal inspectors.

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