US officials have rejected a drug touted as a treatment for Alzheimer’s disease.
“The panel of outside experts for the Food and Drug Administration (FDA) agreed that a pivotal study in patients failed to show ‘strong evidence’ that the drug worked,” reports the Associated Press.
“Alzheimer’s treatment is a huge, urgent, unmet need,” admitted panellist Dr Joel Perlmutter, of the Washington University School of Medicine. “But if we approve something with data that is not strong, we have the risk of delaying good, effective treatments.”
The FDA is not bound by the finding on the candidate called aducanumab, put forward by the company Biogen.
The FDA has until March 2021 to deliver its verdict but sciencemag.org says it typically follows the directives of its independent advisers.
Biogen halted trials of the drug in March 2019 after disappointing interim analysis of patient data. It then proceeded to seek FDA approval in October 2019, shocking the industry. There has been no new drug treatment for Alzheimer’s in 17 years and sciencemag.org contends anything that can slow cognitive decline “would likely bring in tens of billions of dollars in sales”.
Biogen’s stock price rose by 40 per cent just prior to the finding, after documents considered favourable to the drug were posted by the FDA.
“It’s the right decision,” says Robert Howard, a psychiatrist at University College London who has run clinical trials of potential Alzheimer’s treatments and didn’t participate in the meeting. “Approval of aducanumab would be really bad for my field – and really bad for the patients,” he says. “We’ve got to keep a foot on the gas to find a treatment that works … with much more spectacular effect sizes.”
The decision came after a seven-hour virtual meeting last Friday. The New York Times said the panel showed “pointed scepticism”, while a presentation by Dr Billy Dunn, director of the Food and Drug Administration’s office of neuroscience, claimed “the evidence supporting its approval appears strong”.
Ten of 11 panel members disagreed strongly and the other remained uncertain.
“There are literally a dozen different red threads that suggest concerns about the consistency of evidence,” said one member, Dr Caleb Alexander, a professor of epidemiology and medicine at the Johns Hopkins Bloomberg School of Public Health. He said he could not understand “how the FDA could conclude that there is substantial evidence of effectiveness”.
Approximately 30 million people globally have Alzheimer’s and that number is expected to double by 2050.
Samantha Budd Haeberlein, a Biogen senior vice-president and head of neurodegeneration development, told the panel that “the benefit-risk profile for aducanumab is favourable and potentially prolonged patients’ independence by several months”.
But NBC news reported that the consumer group Public Citizen warned that the expensive drug “could bankrupt our healthcare system” while giving patients false hope.
“It’s a biotech medicine made from living cells, and such drugs are very expensive.”
The New York Times estimated the annual cost of the drug to a patient at $50,000.
Part of the issue for the panel was that a trial with successful results was being favoured over the unsuccessful results of another trial.
“There are currently five medications that have been approved to treat cognitive and memory symptoms, but they typically delay decline for only several months. The last new drug was approved in 2003, and none of them address the specific disease process of Alzheimer’s.
“Perfection may be the enemy of the good, but for aducanumab, the evidence doesn’t even rise to ‘good’,” neurologist Dr David Knopman wrote in a comment submitted to the panel before Friday’s hearing.
Dr Knopman sits on the advisory panel but was recused from the hearing because he worked on the aducanumab trials.
“Contrary to the hope that aducanumab will help Alzheimer patients, the evidence shows it will offer improvement to none, it will harm some of those exposed, and it will consume enormous resources,” Dr Knopman said.
Do you follow development in trials of drugs for Alzheimer’s. Does the rejection of this drug give you comfort that stringent controls are in place?
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