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 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.
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 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.
'MEANINGFUL EFFICACY'
"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)
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