Promising Alzheimer’s Drug on FDA Fast Track

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Well before Covid-19 emerged, medical researchers warned that the rapid increase of Alzheimer’s disease in older populations would lead to the next “pandemic.”

In many respects, they were right. According to the Alzheimer’s Association, nearly 10 percent of the U.S. population over 65 years old suffers from that progressively destructive brain disorder. The same organization estimates that the percentage afflicted with Alzheimer’s disease will more than double by the year 2050.

Alzheimer’s not only slowly robs its victims of their mental faculties, it is also surprisingly deadly. The National Institute of Aging ranks Alzheimer’s disease as the sixth leading cause of death in the United States. Other estimates have it ranked as high as the third leading cause of death, trailing only heart disease and cancer.

Sadly, medical science has struggled to provide solutions for those suffering from Alzheimer’s. The last drug approved to combat the disease occurred back in 2009, and currently available treatments can at best only slow the progress of the disease.

Fortunately, a drug named aducanumab provides cause for optimism.

Developed by drug producers Biogen and Eisai, aducanumab was set to become the first new drug to help treat Alzheimer’s disease this decade. Its producers, however, abandoned their hope of getting FDA approval after aducanumab failed a futility test in March 2019. Eight months later, Biogen developed an expanded dataset to examine the drug’s impact on patients taking larger doses. Astonishingly, researchers found aducanumab curtailed the effects of Alzheimer’s disease.

The discovery prompted Biogen and Eisai to request the U.S. Food and Drug Administration to allow aducanumab to re-enter the agency’s approval process. The request was highly irregular, considering the drug had only produced one positive trial, and the evidence for its effectiveness was compiled using an unconventional method for the FDA.

Despite widespread controversy, the FDA made an exception and allowed aducanumab to continue through the approval process. Most recently, aducanumab was selected for priority review with an anticipated approval coming in March 2021.

Aducanumab’s unlikely story, from an abandoned project to a first-of-its-kind treatment for Alzheimer’s disease, contains a critical lesson for future drug approval policy. As one Fierce Biotech article explains:

How the FDA rules on aducanumab will show how far the FDA and its commissioner, Stephen Hahn, M.D., are willing to diverge from its established approval standards. Under U.S. law, companies need to show “substantial” evidence of effectiveness to win approval.

The FDA has long vowed to incorporate different forms of evidence, including big data analysis and other approaches into its approval process. While the agency has made exceptions to its standards and approved drugs out of expediency, it has yet to make systematic changes to expand its definition of what should be considered “substantial evidence.” Let’s hope aducanumab motivates this desperately needed reform.