On June 7, the Food & Drug Administration approved Aduhelm (aducanumab) to treat Alzheimer's patients, making it the first new drug approval for a disease impacting more than six million Americans in almost 20 years.
While the Alzheimer's Association strongly supported FDA approval, citing a 22 percent reduction in cognitive and functional decline in clinical trials, the drug by Biogen Inc. was approved without the recommendation of the Peripheral and Central Nervous System Drugs Advisory Committee. By mid-June, three members of the advisory panel had resigned in response to the controversial approval.
In an article outlining the FDA's process, Patrizia Cavazzoni, MD, director of the FDA Center for Drug Evaluation and Research, noted Aduhelm's late-stage development program consisted of two phase 3 clinical trials where one study met the primary endpoint in showing reduction in clinical decline but the second trial did not meet that endpoint.
"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," Cavazzoni wrote. "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."
In response to the uproar, the Alzheimer's Association reaffirmed their support, noting their research experts and advisors are deeply familiar with the science that led to the approval and agreed with the decision. The organization also called on the confirmatory trial that was part of the approval process to begin promptly.
The statement further read: "Our focus has and will continue to be access to this treatment for all likely to benefit. Approval is the fundamental first step to access. The first drug in a category invigorates the field, increases investments in new treatments and encourages greater innovation.
"Following approval, the manufacturer, Biogen, announced their intention to price Aduhelm at $56,000 per year. This price is simply unacceptable. For many, this price will pose an insurmountable barrier to access, it complicates and jeopardizes sustainable access to this treatment, and may further deepen issues of health equity. We call on Biogen to change this price."