The Food and Drug Administration (FDA) on Monday approved what it described as a “first-of-its-kind” treatment for Alzheimer’s disease, which it says “targets the fundamental pathophysiology of the disease.”
The approved drug, aducanumab, is what the Washington Post described as the “first drug cleared that is designed to alter the course of the disease by slowing the deterioration of brain function — not just to ease symptoms.”
“Aduhelm was approved using the accelerated approval pathway, which can be used for a drug for a serious or life-threatening illness that provides a meaningful therapeutic advantage over existing treatments,” the FDA said in a Monday statement:
We recognize the unmet medical need for patients with Alzheimer’s disease, the devastating nature of the disease for patients and their families, and the urgency to make treatment available.
— U.S. FDA (@US_FDA) June 7, 2021
Researchers evaluated Aduhelm’s efficacy in three separate studies representing a total of 3,482 patients. The studies consisted of double-blind, randomized, placebo-controlled dose-ranging studies in patients with Alzheimer’s disease. Patients receiving the treatment had significant dose-and time-dependent reduction of amyloid beta plaque, while patients in the control arm of the studies had no reduction of amyloid beta plaque.
These results support the accelerated approval of Aduhelm, which is based on the surrogate endpoint of reduction of amyloid beta plaque in the brain—a hallmark of Alzheimer’s disease. Amyloid beta plaque was quantified using positron emission tomography (PET) imaging to estimate the brain levels of amyloid beta plaque in a composite of brain regions expected to be widely affected by Alzheimer’s disease pathology compared to a brain region expected to be spared of such pathology.
The approval is significant, as no treatment has been approved in about 18 years, since 2003. However, the approval does not come without controversy, given what has been described as its “complicated” history.
Per the Post:
Supporters of the medication have predicted that FDA approval would stimulate newfound interest and investment in researching therapies for the progressive, terminal illness. Biogen says the medication, by slowing the disease, gives patients valuable time to be with their families and to perform everyday tasks such as cleaning and shopping.
But critics have argued that data on the drug’s effectiveness is weak and that FDA approval represents a dangerous lowering of standards in response to pressure from patients and advocacy groups.
The intravenous treatment does not cure Alzheimer’s disease or reverse it. And it could carry a price tag as high as $50,000 a year per patient, making it a blockbuster product for the company and adding billions of dollars to the nation’s health tab.
“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,” Patrizia Cavazzoni, M.D., director of the FDA’s Center for Drug Evaluation and Research, said in a statement.
Cavazzoni described the treatment option as 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,” Cavazzoni added.