Alzheimer’s: U.S. Regulatory Authority Approves Use of “Aducanumab” on the Condition of Further Studies

Bonn, June 8th, 2021. The U.S. Food and Drug Administration (FDA) has approved the use of the Alzheimer’s drug “aducanumab” (brand name is “Aduhelm”) under the condition that further studies are conducted to verify its clinical benefit. The marketing authorization had been filed by the pharmaceutical companies Biogen and Eisai. This is the first time the FDA approves a potential treatment for Alzheimer’s disease primarily based on biomarker data. Aducanumab targets proteins associated with the disease. How the drug will perform in clinical routine, remains to be seen. Whether it will be deployed in Germany depends on the European regulatory authority EMA, whose decision is still pending.

Statement by Prof. Pierluigi Nicotera, Scientific Director and Chairman of the DZNE’s Executive Board:

“Millions of people around the world with Alzheimer’s disease and their families are hoping for better treatment for this devastating condition. So far, clinical data on the effect of aducanumab on cognitive decline due to Alzheimer’s disease have been inconclusive. However, opening the possibility to treat patients with Aduhelm will likely produce solid efficacy data. This would open up new therapeutic possibilities. It is remarkable that the FDA recognizes the urgent need to treat dementia by approving the use of Aduhelm because of biomarker-based evidence.”

General background

The FDA’s decision is a game changer, as it the first time that an Alzheimer’s drug is approved primarily on the basis of biomarkers, i.e., biomolecular criteria, rather than clinical data. The FDA’s decision could have an impact on future clinical trials and marketing authorization decisions.

Treatment with aducanumab is expected to be complex and costly, requiring regular infusion of the drug as well as monitoring of the brain on a regular basis. Another challenge is identifying those patients who are suitable for treatment. Participants in clinical studies of aducanumab were selected on the basis of complex analyses, including examinations of the brain using positron emission tomography. This technique is not widely available, but mainly at university hospitals. Therefore, it will be necessary to develop decision-making criteria that can be used in everyday medical practice.

Several other similar drugs, as well as a number of other approaches to combat Alzheimer's disease, are currently being investigated. Effective treatment of the disease will likely require combination therapy. Thus, the FDA’s decision could be a first step toward better treatment, but there is certainly a long way to go.

Background on aducanumab

Aducanumab (brand name is “Aduhelm”) is a genetically engineered antibody that binds to amyloid-beta proteins. In individuals with Alzheimer’s disease, these proteins accumulate in the brain and form aggregates – called “plaques” – in the vicinity of neurons. Due to this phenomenon and other evidence, amyloid-beta proteins are considered to be important protagonists in the disease process. Clinical trials in which aducanumab was administered by infusion showed that this treatment significantly reduced the amount of plaques – which served as biomarkers – in the brain. In one study, there was also evidence that the treatment could slow mental decline to some extent in people with early Alzheimer’s disease, but a second study could not demonstrate this effect. Given these conflicting findings, there was controversy about the effectiveness of aducanumab in the run-up to the FDA’s decision. In a statement on June 7 the FDA announced: “Although the Aduhelm data are complicated with respect to its clinical benefits, FDA has determined that there is substantial evidence that Aduhelm reduces amyloid beta plaques in the brain and that the reduction in these plaques is reasonably likely to predict important benefits to patients.”

In the same statement the FDA noted that the responsible drug companies will be required to conduct studies to verify the clinical benefit. Failure to confirm this benefits could lead to removing the drug from the market.

Aducanumab was jointly developed by Biogen and Eisai. The two pharmaceutical companies have also filed for marketing authorization of this drug in Europe and Japan. Decisions of the responsible authorities are still pending.

Background on Alzheimer’s disease

According to estimates, up to two-thirds of all people with dementia are affected by Alzheimer’s disease, it thus considered to be the most common form of dementia. Alzheimer’s is associated with a gradual loss of neurons in the brain, resulting in a decline in mental abilities. Affected persons in the advanced stages of the disease depend on care and assistance from others. Current medications and non-pharmacological therapies can alleviate the symptoms of Alzheimer’s to some extent; however, they do not address the causes of the disease and can therefore neither stop nor delay its progression – and especially they cannot repair nerve damage that has already occurred. Aducanumab is the first drug to reach the market that targets mechanisms of the disease.

According to estimates, there are about 1.6 million individuals with dementia in Germany, and about 50 million worldwide.

About Deutsches Zentrum für Neurodegenerative Erkrankungen (German Center for Neurodegenerative Diseases, DZNE): The DZNE is a research center that addresses all aspects of neurodegenerative diseases (such as Alzheimer’s, Parkinson’s and Amyotrophic lateral sclerosis) in order to develop novel approaches of prevention, treatment, and health care. It is comprised of ten sites across Germany and cooperates closely with universities, university hospitals, and other research institutions on a national and international level. The DZNE is publicly funded and a member of the Helmholtz Association of German Research Centers.

Media relations

Dr. Marcus Neitzert
+49 228 43302-267

Further information

FDA Statement, 6/7/2021

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