JAMA Sub-Journal: Why did the FDA accelerate the approval of Aducanumab, a new drug for Alzheimer's disease?

Alzheimer's disease is the sixth leading cause of death in the United States.

On June 7, 2021, the U.

Billy Dunn et al.

The reason for the accelerated approval is to provide drugs for the treatment of serious diseases as soon as possible when the final clinical benefits of the drugs are still uncertain at the time of approval

(1) The drug must be used to treat serious diseases for which medical needs are not met;

(2) Compared with existing therapies, the drug is expected to provide meaningful clinical advantages;

(3) The effect of the drug on the surrogate endpoint must be shown; the surrogate endpoint, as an effective indicator to measure clinical outcomes and reduce clinical observation time, has begun to be widely used in the design of new drug clinical trials, and it has gradually become widely accepted by the FDA for reviewing new drugs

(4) There is a reasonable possibility to predict the clinical benefit of the drug

Aduhelm meets the above conditions

The main risk of the drug is the increased incidence of Aβ-related imaging abnormalities in patients receiving Aduhelm treatment

In magnetic resonance imaging, brain amyloid abnormalities can be observed with edema or hemochromatosis (signs of microbleeds and hemochromatosis), usually (76%) asymptomatic

As the clinical benefits of Aduhelm are still uncertain, as a project for accelerated approval, Biogen is required to verify the benefits of the drug

When weighing the benefits and risks of Aduhelm , consider that Alzheimer's disease can lead to irreversible loss of memory, cognition and daily activities

Billy Dunn et al.