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The Food and Drug Administration (FDA) has revised its recommendation of who should receive Aduhelm, the controversial Alzheimer’s disease drug approved by the FDA last month.

The original label stated the treatment was for anyone with Alzheimer’s disease, encompassing six million Americans. Under the new label, the FDA recommends Aduhelm to “patients with mild cognitive impairment or mild dementia stage of disease,” reflecting the population tested in clinical trials and cutting the number of eligible Americans to about two million.

“The revision of this label is yet another piece of evidence that should cause the American public to be concerned about how FDA is practicing its regulatory science,” PMC co-director Dr. Jason Karlawish told the New York Times.

Dr. Jason Karlawish, co-director of PMC, was quoted by The Washington Post regarding the negative potential consequences of the Food and Drug Administration’s approval of Aduhelm.

“Companies are companies, I can’t fault them for doing what companies do, but we have now unleashed drugs that we are not sure what the benefits are, and that could have enormous consequences,” said Dr. Jason Karlawish.

This past Wednesday, the Penn Memory Center held a community event for PMC patients and families led by co-directors Drs. Jason Karlawish and David Wolk to discuss the Food and Drug Administration’s (FDA) decision to approve Aduhelm (aducanumab). The focus was the impact of the approval on the PMC’s clinical and research operations. 

By Dr. Patrizia Cavazzoni, Director, FDA Center for Drug Evaluation and Research

Today FDA approved Aduhelm (aducanumab) to treat patients with Alzheimer’s disease using the Accelerated Approval pathway, under which the FDA approves a drug for a serious or life-threatening illness that may provide meaningful therapeutic benefit over existing treatments when the drug is shown to have an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients and there remains some uncertainty about the drug’s clinical benefit.

This approval is significant in many ways. Aduhelm is the first novel therapy approved for Alzheimer’s disease since 2003. Perhaps more significantly, Aduhelm is the first treatment directed at the underlying pathophysiology of Alzheimer’s disease, the presence of amyloid beta plaques in the brain.  The clinical trials for Aduhelm were the first to show that a reduction in these plaques—a hallmark finding in the brain of patients with Alzheimer’s—is expected to lead to a reduction in the clinical decline of this devastating form of dementia.

We are well-aware of the attention surrounding this approval. We understand that Aduhelm has garnered the attention of the press, the Alzheimer’s patient community, our elected officials, and other interested stakeholders. With a treatment for a serious, life-threatening disease in the balance, it makes sense that so many people were following the outcome of this review. Further, the data included in the applicant’s submission were highly complex and left residual uncertainties regarding clinical benefit. 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.

At the end of the day, we followed our usual course of action when making regulatory decisions in situations where the data are not straightforward. We examined the clinical trial findings with a fine-tooth comb, we solicited input from the Peripheral and Central Nervous System Drugs Advisory Committee, we listened to the perspectives of the patient community, and we reviewed all relevant data. We ultimately decided to use the Accelerated Approval pathway—a pathway intended to provide earlier access to potentially valuable therapies for patients with serious diseases where there is an unmet need, and where there is an expectation of clinical benefit despite some residual uncertainty regarding that benefit. In determining that the application met the requirements for Accelerated Approval, the Agency concluded that the benefits of Aduhelm for patients with Alzheimer’s disease outweighed the risks of the therapy.

Click the headline or the button below to read this announcement on the FDA website.

Amyloid plaque in the brain. Photo: Adobe

In a recent essay published in STAT News, PMC co-director Dr. Jason Karlawish argued that, given aducanumab’s “incomplete and contradictory” data and the results of an analysis of aducanumab’s effectiveness and value by the Institute for Clinical and Economic Review, the FDA should not approve the drug at this time and stated he will not prescribe it to his patients.

Click the headline or the button below to read this article on STAT News.