The Food and Drug Administration approved a new drug for Alzheimer’s disease Monday, purportedly giving doctors a new but much-debated method of directly addressing the Alzheimer’s disease process instead of merely treating the symptoms of it.
The drug, called aducanumab, was approved through the FDA’s accelerated approval program, which is reserved for drugs that “fill an unmet medical need based on a surrogate endpoint” but that do not definitively improve patient outcomes. As Stat News notes, this means that, while the drug has shown it can eliminate the toxic protein clumps experts believe contribute to dementia, the regulatory agency doesn’t know definitively if it actually helps improve cognition in the patients who receive it.
“Alzheimer’s disease is a devastating illness that can have a profound impact on the lives of people diagnosed with the disease as well as their loved ones,” said Patrizia Cavazzoni, M.D., director of the FDA’s Center for Drug Evaluation and Research. “Currently available therapies only treat symptoms of the disease; this treatment option is the first therapy to target and affect the underlying disease process of Alzheimer’s. As we have learned from the fight against cancer, the accelerated approval pathway can bring therapies to patients faster while spurring more research and innovation.”
According to The New York Times, both proponents and critics of the drug, which also goes by the brand name Aduhelm, agree that it “substantially reduces levels of amyloid,” a protein that “clumps into plaques in the brains of Alzheimer’s patients and is considered a biomarker of the disease.” But in the past, drugs that have reduced amyloid levels in patients have still failed to improve outcomes in patients.
“There’s so little evidence for effectiveness,” said Dr. Lon Schneider, director of the California Alzheimer’s Disease Center at the University of Southern California, who helped conduct one of the aducanumab trials, told the New York Times. He added: “I don’t know what caught the F.D.A.’s fancy here.”
But Harry Johns, chief executive of the Alzheimer’s Association, called the approval a “victory for people living with Alzheimer’s and their families.”
“This is the first FDA-approved drug that delays decline due to Alzheimer’s disease,” said Johns in a statement. “This means individuals may have more time to actively participate in daily life, have sustained independence and hold on to memories longer. We can experience longer — the relationships we hold most dear — our families and friends.”
From Stat News:
For decades, scientists who study Alzheimer’s have focused much of their research on amyloid. A succession of companies including Eli Lilly, Pfizer, Roche, and Merck have attempted to treat Alzheimer’s with various types of antibody drugs that work by targeting and eliminating beta-amyloid — all without success.
Biogen believed it could succeed where the others failed; it designed Aduhelm to be more potent and therefore better able to eliminate amyloid plaques than earlier drugs. The company also focused on administering Aduhelm to people with mild cognitive impairment or the earliest signs of Alzheimer’s, before brain cells were too damaged to stem the tide of the disease.
An FDA advisory committee voiced opposition to the drug’s approval in November. However, the FDA isn’t required to listen to the committee.
Biogen, the maker of the drug, has estimated it will list at $56,000 per year — which does not necessarily reflect the actual price patients or insurance will pay. According to Stat News, analysts believe the price will be between $10,000 and $25,000 per year for patients. The drug is administered intravenously once every four weeks.
Michel Vounatsos, CEO of Biogen, defended the price of the drug Monday, reportedly telling CNBC that the list price was “fair.” He also called the drug the product of “two decades of innovation.” The last Alzheimer’s drug was approved by the FDA in 2003.
“We feel a great sense of purpose and responsibility to turn the hope of today’s FDA approval of [Aduhelm] into a reality for people living with Alzheimer’s disease and their families,” said Alisha Alaimo, President of Biogen U.S. “We are committed to access and health equity as top priorities and will continue working with multiple stakeholders with the goal of helping patients who may benefit from treatment obtain care as quickly as possible.”
Under the FDA accelerated approval rules, the drug-maker will still need to conduct stage IV clinical trials to prove that Aduhelm provides a clinical benefit to Alzheimer’s patients. “If the confirmatory trial shows that the drug actually provides a clinical benefit, then the FDA grants traditional approval for the drug,” notes the FDA. “If the confirmatory trial does not show that the drug provides clinical benefit, FDA has regulatory procedures in place that could lead to removing the drug from the market.”