'Historic' Decision Expected on U.S. Approval of Alzheimer's Drug
The first drug ever shown to slow the progression of Alzheimer's disease could be approved by the U.S. Food and Drug Administration on Monday, but experts say that approval will be surrounded by controversy.
In clinical trials, aducanumab showed a 22% reduction in the development of thinking and memory problems associated with Alzheimer's disease, according to a statement from the Alzheimer's Association.
The association has urged the FDA to approve the drug, hailing it as a treatment "that could make a meaningful difference in the lives of individuals living with Alzheimer's and their families."
However, an approval would fly in the face of the FDA's independent advisory committee, as well as a number of prominent Alzheimer's experts.
Ten of the FDA panel's 11 members voted against approval in November, even after hearing a presentation by the agency's director of neuroscience arguing there is "strong" evidence supporting the drug.
Three advisory committee members went even further, writing a detailed critique of the evidence. They have been joined by a host of other scientists, including even those who participated in aducanumab's clinical trials, The New York Times reported.
"I simply don't see a path for approval because of the absence of evidence that's been shared to date that this product works, and I think it would set a remarkably dangerous precedent - not only for the field of Alzheimer's research but also for the broader regulation of prescription drugs in our country," Dr. G. Caleb Alexander, an FDA advisory committee member with the Johns Hopkins Bloomberg School of Public Health in Baltimore, told the Times.
Aducanumab, developed by the company Biogen, targets the sticky protein amyloid-beta, which forms plaques in the brains of early-stage Alzheimer's patients.
It's being hailed by advocates like the Alzheimer's Association as the first drug to directly target the cause of the progressive neurological disease, rather than just its symptoms. If approved, it would be the first new Alzheimer's drug approved by the FDA since 2003.
"This will give us a new biological foothold to build on," Stephen Salloway, director of neurology and the Memory and Aging Program at Butler Hospital in Providence, R.I., told the Washington Post. "To get the best in class, you have to have the first in class."
The intravenous drug would be a moneymaker for Biogen, and could cost as much as $50,000-a-year per patient, the Post reported.
The drug's development path has been rocky. In March 2019, Biogen announced it was halting two clinical trials of aducanumab because the drug didn't seem to be making enough of a difference.
But Biogen reversed its decision seven months later, after more analysis of data from one of the trials found aducanumab showed some positive effect at higher doses, the Times reported.
Dr. David Knopman, a neurologist at the Mayo Clinic and a principal investigator for one of the trials, told the Times, "One study was positive, and one identically performed study was negative. I don't think it takes a PhD in statistics to see that that's inconclusive."
Experts also noted that the drug poses potential safety risks for some patients. About two of five patients taking the high dose of aducanumab experienced brain swelling or bleeding, the Times said, but the effects were mild enough that they caused only 6% of participants to drop out of the trial.
Advocacy groups urging approval noted the need for some new treatment, even if its effectiveness hasn't been conclusively proven.
"With more than 6 million Americans living with Alzheimer's, the need for a treatment for a disease that has no survivors has never been greater," the Alzheimer's Association said in its statement.
The Alzheimer's Association has more about FDA-approved treatments for Alzheimer's symptoms.
SOURCES: June 2, 2021, statement, Alzheimer's Association; The New York Times; Washington Post