June 7 (Reuters) - The U.S. FDA on Monday is expected to give its thumbs up or down on Biogen Inc’s aducanumab, a decision that will affect the future of Alzheimer’s disease research and treatment and show the agency’s willingness to approve badly needed medicines based on less than definitive data.
Aducanumab has been at the center of a divisive public battle. The Food and Drug Administration is under enormous pressure from patient advocacy groups and some doctors to approve the first major drug for the mind-wasting condition, while many experts say huge amounts of money will be spent despite a lack of compelling evidence aducanumab can provide meaningful benefit.
Approval could reinvigorate a failure-laden field that has been abandoned by many large drugmakers and give Biogen a new multibillion-dollar seller. Its shares rose 5% on Friday ahead of the expected decision.
A rejection would likely spell the end of aducanumab and possibly sound the final death knell for similar experimental Alzheimer’s drugs in development. The FDA decision could also have wider ramifications, analysts said.
It is “likely to reverberate throughout the biopharma sector, influencing overall sentiment on the space, perceptions on regulatory flexibility, and business development dynamics,” RBC Capital Markets analyst Brian Abrahams said in a recent research note.
Alzheimer’s disease is the sixth leading cause of death in the United States. There is “a profound and enormous unmet medical need” for new treatments, Billy Dunn, director of neurology products evaluation for the FDA, said at a November advisory committee meeting.
Aducanumab aims to remove sticky deposits of a protein called amyloid beta from the brains of patients in earlier stages of Alzheimer’s to stave off the ravages of memory loss.
The amyloid theory has been at the center of Alzheimer’s research for many years with a long list of failures to show for it, and seemed on life support until Biogen revived its aducanumab program.
Biogen’s drug has been hailed by patient groups and some neurologists eager for an effective option to treat the lethal disease, but trial results have been inconsistent.
One Phase III aducanumab study resulted in a statistically significant 23% benefit at slowing declines in cognition and daily function compared to a placebo.
However, a second large study failed to show that benefit, leading Biogen and partner Eisai Co to scrap development in March 2019.
They reversed course in October, saying a more detailed company analysis of the failed study showed that some patients with very early forms of the disease benefited from taking higher doses over an extended period of time.
That was enough for some advocates desperate for anything that might help. Opponents say the FDA risks setting a precedent for lowering its standards, opening a path to expensive, potentially ineffective drugs by not requiring truly compelling evidence for approval.
The waters were further muddied when FDA staff concluded that the successful trial offered enough persuasive evidence, only to have a panel of outside advisers to the agency vote in November that data did not prove aducanumab could slow Alzheimer’s progression.
Advocates have been marshalling support since.
“We believe that the perspective of the panel was too narrow ... We conclude that aducanumab achieves the standard of meaningful efficacy with adequate safety,” six prominent Alzheimer’s experts, including lead author Dr. Jeffrey Cummings, director of the University of Nevada Las Vegas Chambers-Grundy Center for Transformative Neuroscience, wrote last month in Alzheimer’s Research & Therapy.
Previous clinical studies of Alzheimer’s drugs had largely targeted patients in later stages of the disease, while many experts now believe attacking it as early as possible may be the key to success.
Aducanumab was studied in patients with early disease who test positive for a component of amyloid brain plaques. Some trial patients experienced potentially dangerous brain swelling.
Biogen has estimated that around 1.5 million Americans would be eligible for treatment with aducanumab, which is given by monthly infusion, raising concerns about costs to the healthcare system for both diagnostics and treatment.
If approved, the drug could reap billions of dollars in sales, mostly at a cost to the U.S. government’s Medicare health insurance program for seniors.
The Institute for Clinical and Economic Review, an influential drug pricing research group, in a May report cited “insufficient” evidence that aducanumab provides a net health benefit. It said data so far indicate a cost-effective price of no more than $8,300 per year. Looking only at favorable trial results, that price rises as high as $23,100, ICER said.
The research group put the fair price for any drug proven to halt progression of Alzheimer’s-related dementia at $50,000 to $70,000 per year. (Reporting by Deena Beasley in Los Angeles; Editing by Bill Berkrot)