Everything To Know About The New Controversial FDA-Approved Alzheimer’s Drug

On Monday, the Food and Drug Administration (FDA)Trusted Source announced accelerated approval for drugmaker Biogen’s Aduhelm, the brand name for aducanumab. It is the first drug to treat a likely cause of Alzheimer’s disease, despite controversy around whether clinical evidence proves it works.

While other drugs relieved some symptoms of the condition, Aduhelm has been found in one clinical trial to actually slow the progression of plaques associated with Alzheimer’s in patients with early stages of the disease.

But the drug’s approval is also controversial, as it is not clear how beneficial it will be at stopping the clinical symptoms of the disease.

Amyloid plaques associated with Alzheimer’s disease
“In Alzheimer’s disease, nerve cell death is due to the deposit of amyloid plaques and neurofibrillary tangles in the brain, and that eventually leads to clinical dysfunction,” Dr. Gayatri Devi, a neurologist at Lenox Hill Hospital in New York who specializes in memory disorders, told Healthline.

According to Devi, author of “The Spectrum of Hope: An Optimistic and New Approach to Alzheimer’s Disease and Other Dementias,” a similar process can occur with other forms of dementia.

“For example, in Lewy body disease [a common form of dementia], there are Lewy body deposits,” she said, adding, “There is data that shows that removing or preventing plaques can help in Alzheimer’s disease.”

Aduhelm targets underlying disease process
“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,” Dr. Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research, said in a statementTrusted Source.

She emphasized that while currently available therapies only treat symptoms of Alzheimer’s, Aduhelm is the first therapy to target and affect the underlying disease process of Alzheimer’s.

“As we have learned from the fight against cancer,” she said, “the accelerated approval pathway can bring therapies to patients faster while spurring more research and innovation.”

According to the FDA, this is the first new treatment approved for Alzheimer’s since 2003.

According to the FDA, Biogen researchers evaluated Aduhelm’s efficacy in two different studies that looked at almost 3,500 patients.

All three were “double-blind, randomized, placebo-controlled, dose-ranging studies” in patients living with Alzheimer’s disease.

Researchers found that patients who received the drug had significant dose- and time-dependent reduction of amyloid beta plaque. But patients in the control arm of the studies, or those not given Aduhelm, had no reduction of this plaque.


Drug’s approval is controversial
The findingsTrusted Source of the studies didn’t clearly show that people were likely to have fewer symptoms of Alzheimer’s disease if they were taking the drug. In one of the studies, researchers found a small benefit for people taking the drug. In the other study, however, no benefit was seen.

The FDA has asked that Biogen now start another trial to verify that the drug actually works. If that trial fails, the FDA could revoke approval.

According to the FDA, not all experts agreed that Aduhelm merited accelerated approval.

“The data included in the applicant’s submission were highly complex and left residual uncertainties regarding clinical benefit,” said the FDA in a statementTrusted Source. “There has been considerable public debate on whether Aduhelm should be approved. As is often the case when it comes to interpreting scientific data, the expert community has offered differing perspectives.”

The FDA confirmed that it examined the clinical trial findings with a “fine-tooth comb,” solicited input from the Peripheral and Central Nervous System Drugs Advisory CommitteeTrusted Source, reviewed all relevant data, and “listened to the perspectives of the patient community” before making their decision.