FDA panel upholds highly controversial ALS drug in rare second review

A panel of federal health advisers voted Wednesday to recommend approval of an experimental drug to treat Lou Gehrig’s disease.

Food and Drug Administration advisers voted 7 to 2 that the data from Amylyx Pharma required approval, but there were ongoing concerns about the strength and reliability of the company’s only study. Voting is non-binding and FDA expects to make a final decision by the end of the month.

Despite the negative reviews released by FDA internal scientists in advance of the meeting, the majority of external panelists said they presented sufficient evidence to suggest that Amylyx could possibly help patients live longer. .

Dr. Liana Apostolova of the Indiana University School of Medicine, who voted for approval, said: “It wasn’t so clear at the last meeting and I’m still skeptical.”

Amylyx also appears to be profiting from an unusual exchange in which company executives have promised to withdraw the drug from the market at the FDA’s request if large ongoing studies do not confirm its benefits. It looked like

“We have some confidence that if approval is issued, it can be withdrawn in the future,” Apostolova said.

Wednesday’s vote ended a rare second meeting to review several new statistical analyzes submitted by Amylyx to support the benefits of treatments that slow disease progression and extend life. The same group of neurology experts narrowly voted against the drug in March over concerns about missing data and implementation errors in the company’s research.

The FDA has approved only two drugs to treat amyotrophic lateral sclerosis (ALS), which destroy nerve cells necessary for basic functions such as walking, talking and swallowing.

ALS patients and their families rallied behind Amylyx’s drug and began an aggressive lobbying campaign to rally members of Congress to seek approval from the FDA.

ALS drug reviews are being watched as an indicator of the FDA’s flexibility and ability to withstand external pressure when reviewing experimental drugs for terminally ill patients.

Dr. Billy Dunne, FDA’s chief neurology review officer, opened the meeting by detailing “concerns and limitations” of the Amylyx data, emphasizing the need for new treatment options.

“We are very sensitive to the urgent need to develop new treatments for ALS,” said Dunn.

Dunn also noted that larger Amylyx studies being conducted in the US and Europe could provide “more conclusive results” by 2024.

In a highly unusual move, Dunn says agencies will approve the drug more aggressively if Amylyx promises to stop the drug if it doesn’t work in an ongoing 600-patient trial. He then asked the company’s co-founders to publicly commit to that step, and Amylyx co-CEO Justin Klee said in that scenario the company could voluntarily take the drug. said it would withdraw the

Although the FDA has the power to force companies to withdraw drugs from the market, it is generally quicker for pharmaceutical companies to do so voluntarily. If companies resist removal, the regulatory process can last for years.

Dr. Caleb Alexander of Johns Hopkins University said he was one of two panelists who voted against the drug.

Amylyx has conducted one small, intermediate-stage trial of its drug, which has shown efficacy in slowing disease progression, but FDA reviewers say it has been plagued by missing data and other problems. I was.

FDA statistician Tristan Massie told panelists, “The final results of a single study are borderline and not statistically convincing.

A company in Cambridge, Massachusetts, says follow-up data collected after the study ended showed that the drug extended life. , survived about 10 months longer than patients who did not take the drug.

Panellists in favor of the drug cite the data and the drug’s mild side effects, suggesting that even if it doesn’t ultimately slow ALS, it poses little downside for patients.

Dean Forman, a biostatistician at the National Institutes of Health, said, “This drug is not harmful and appears to work. There are no indications of its safety.

Earlier Wednesday, more than 20 ALS researchers, patients, and family members told advisors they supported the approval. The agency also received over 1,200 written comments, mostly from her ALS patient advocates.

“I know it works, so I’m asking you for approval,” said Greg Canter, who was diagnosed with ALS in 2018 and participated in the Amylyx study. , believed that the drug improved lung capacity and slowed decline.

Amylyx medicine comes as a powder that combines two old medicines. One is a prescription drug for liver disorders and the other is a dietary supplement used in traditional Chinese medicine.

At stake is the FDA’s approval last year of the controversial Alzheimer’s drug Aduhelm, which was reviewed by the same agency’s scientists and outside advisors.

In that case, the FDA ignored overwhelming negative votes by outside advisors, three of whom resigned because of the decision. The agency’s approval, which followed an unscheduled meeting with pharmaceutical company Biogen, is under investigation by Congress and federal inspectors.

Source link

Leave a Reply

Your email address will not be published. Required fields are marked *