Ice Bucket Challenge-Funded Medication To Be Reviewed By FDA Advisers:
Advisory board members for the Food and Medicine Administration will vote on whether to approve an experimental drug for ALS patients on Wednesday.
Experts say the stakes are high for the agency, which is under pressure from patients and advocacy organizations to approve the medicine fast, citing a major unmet need for patients, while also facing heavy scrutiny from the scientific community over its previous decisions.
Lou Gehrig’s disease, often known as amyotrophic lateral sclerosis (ALS), is a progressive and deadly neurological illness.
The medicine, developed by Amylyx Pharmaceuticals of Cambridge, Massachusetts, is intended to delay the disease’s course by preventing the premature death of cells that transport messages from the brain to muscle cells.
However, in its application to the FDA for approval, the business is relying solely on data from a single clinical trial, a decision that has some health professionals questioning if the corporation has enough proof to establish that the treatment works.
The FDA released a negative analysis of the medicine on Monday, saying in briefing documents that the company’s findings “may not be sufficiently persuasive” to warrant approval.
The Peripheral and Central Nervous System Drugs Advisory Committee’s vote in support of the drug on Wednesday would only be a suggestion, not an official approval, from the agency.
The FDA, on the other hand, usually takes its committees’ suggestions.
Some have linked the FDA’s approach to Amylyx to that of Biogen’s Alzheimer’s medicine Aduhelm.
Even though the advisory council recommended against it, the FDA gave Aduhelm full approval in June.
Outside specialists slammed the FDA’s decision due to the lack of evidence that it helped patients, and at least three members of the advisory group quit in protest after the medicine was approved. Amylyx will be evaluated by the same panel. The FDA’s handling of Amylyx’s medicine has also sparked debate.
It had requested that the firm complete a larger clinical trial before submitting data on the medicine in April of last year, but after meeting with ALS supporters, it reversed course and permitted the company to file for clearance while the additional experiment was still running.
“It’s a crucial case study of the FDA’s capabilities. When you have patients who say, “We want to help,” you have to take responsibility want to try this,” Holly Fernandez Lynch, an assistant professor of medical ethics at the University of Pennsylvania, said of Biogen’s medicine.
Should the FDA approve it despite the lack of certainty?
Patients and advocacy groups like I AM ALS and the ALS Association, who funded a portion of Amylyx’s trial with cash raised from the viral Ice Bucket Challenge in 2014, are excited about the drug’s prospective approval.
Advocacy groups say that if the FDA approves the drug, it will be a significant step forward for the 30,000 people in the United States who have ALS.
The majority of persons with ALS are diagnosed in their 40s to mid-60s, with a two- to five-year survival rate.
According to the Mayo Clinic, the disease’s development varies from person to person, although symptoms usually begin in the hands, arms, legs, or feet before gradually killing cells that govern speech and swallowing.
For the condition and its symptoms, the FDA has approved a few medications, one of which has been demonstrated to extend patients’ lives by around three months.
According to Amylyx, persons who took the medicine lived six months longer than those who received a placebo in a Phase 2 clinical trial with 137 patients.
A three-year follow-up study of the findings revealed that the medication lowered the chance of death by 44%.
With the exception of diarrhoea in the medication group, the reported rates of adverse events were similar among those who received the placebo.
However, FDA experts expressed reservations about the drug’s benefits, stating in briefing documents that Amylyx’s trial results were “not robust.”
The company’s decision to use only one study in its application for clearance also seemed to irritate the agency’s scientists.
A single trial may be enough to prove a drug’s effectiveness in some cases, but Amylyx’s “may not be adequate,” according to the FDA.
According to the records, the FDA also “highly recommended” that the business conduct a Phase 3 trial to assess the drug’s efficacy.
Amylyx declined to comment for this article due to a regulatory review that is still ongoing.
Dr. Stephen Scelsa, a neurologist at Mount Sinai Hospital in New York City, believes the medicine has potential but wants to see more evidence. Scelsa participated in the drug’s trial at Mount Sinai.
Even though the Phase 2 trial showed an apparent improvement, he believes a Phase 3 trial could indicate the medicine doesn’t operate as planned.
“I believe this will work out,” Scelsa remarked. As a scientist, though, I’d like to see Phase 3 results.”
If the FDA approves the drug before the new trial is completed, he says, it will be more difficult to enrol participants for the research because the drug is already available to those who want it.
According to Lynch of the University of Pennsylvania, an FDA “flat out rejection” of the medicine “would surprise me less than I was forecasting last week” after reviewing the FDA materials.
“The FDA has raised some quite severe concerns,” she added, adding that the agency is now in a “terrible position. “All of the possibilities here aren’t great,” she said.
“If the treatment doesn’t work, getting approval is going to be difficult. Refusing to approve it if it works is just as unpleasant — possibly even more so.
The FDA has “regulatory flexibility” to approve medications for “serious diseases with unmet medical needs,” according to the documents released Monday.
It’s still unknown how much influence advocacy groups and patients will have on the FDA’s decision during the public comment session on Wednesday.
The ALS Association’s Brian Frederick emphasised the drug’s popularity, directing NBC News to a petition signed by 50,000 people urging the FDA to approve it as soon as possible.
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