Biogen Updates Prescribing Label of Controversial Alzheimer’s Drug

The saga continues for Biogen’s Alzheimer’s drug Aduhelm as the U.S. Food and Drug Administration (FDA) approved an updated label that emphasizes the disease stages for which the drug was approved.

The label highlights that Aduhelm, formerly known as aducanumab, should only be provided to patients with mild cognitive impairment or mild dementia stage of disease. The updated label notes that there are no safety or effectiveness data on initiating treatment at earlier or later stages of the disease than were studied, Biogen said.

Alfred Sandrock, Jr., head of Research and Development at Biogen, said the company submitted the label update to the FDA with a goal of clarifying the patient population included in clinical studies that supported the regulatory agency’s approval. Sandrock said the updates were based on ongoing conversations with prescribing physicians, patient advocates and the FDA.

“We are committed to continue to listen to the community’s needs as clinical practice adapts to this important, first-in-class treatment option,” Sandrock said in a statement.

Aduhelm was approved by the FDA in June under accelerated approval. The approval was highly controversial due to an overwhelming rejection of the medication by the agency’s advisory committee. Since the approval, at least three members of that committee have resigned in protest. Public Citizen, a watchdog group, has called for additional resignations at the FDA, including acting Commissioner Janet Woodcock due to what it said is a lack of clinical data supporting approval. A U.S. House of Representatives committee is also planning to investigate the approval process, as well as the pricing of the drug, which has a list price of $56,000 per year.

When the FDA approved the drug last month, Dr. Patrizia Cavazzoni, Director of the FDA Center for Drug Evaluation and Research, said Aduhelm is the first treatment directed at the underlying pathophysiology of Alzheimer’s disease, which is 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,” Cavazzoni wrote in a public announcement.

Cavazzoni did note the controversy surrounding the data and ultimate approval of Aduhelm. She said the FDA opted to use the Accelerated Approval pathway, which could allow the agency to demand further clinical data supporting efficacy of the drug. The agency is expected to continue to review real world use of Aduhelm and the FDA is requiring Biogen to conduct a post-approval clinical trial to verify the drug’s clinical benefit.