FDA Approves Updated Label for Controversial Alzheimer’s Therapy

The prescribing information for Aduhelm now specifies its use in patients with a mild form of the disease.

Biogen and Eisai have announced that the FDA has approved updated prescribing information for Aduhelm (aducanumab-avwa) to specify its use in patients with mild Alzheimer’s disease.

The Indications and Usage section of the label now says “Treatment with ADUHELM should be initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was initiated in clinical trials. There are no safety or effectiveness data on initiating treatment at earlier or later stages of the disease than were studied. This indication is approved under accelerated approval based on reduction in amyloid beta plaques observed in patients treated with ADUHELM. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).”

Alfred Sandrock, Jr., M.D., Ph.D., head of research and development at Biogen, said in a statement, “Based on our ongoing conversations with prescribing physicians, FDA and patient advocates, we submitted this label update with the goal to further clarify the patient population that was studied across the three Aduhelm clinical trials that supported approval. We are committed to continue to listen to the community’s needs as clinical practice adapts to this important, first-in-class treatment option.”

The FDA had approved the therapy in June 2021, even though an advisory committee in November 2020 recommended against approval. FDA officials had said in a statement the therapy is the first treatment directed at the underlying pathophysiology of Alzheimer’s disease, the presence of amyloid beta plaques in the brain.

The controversy surrounding this therapy is related to both the clinical trial results and Biogen's pricing of the therapy. The late-stage development program for Aduhelm consisted of two phase 3 clinical trials. One study met the primary endpoint, showing reduction in clinical decline, while the second trial did not meet the primary endpoint.

At the end of June, the Institute for Clinical and Economic Review (ICER) updated its analysis, stating the price range needed to reach standard cost-effectiveness thresholds is between $3,000 and $8,400 a year, compared with the annual price of $56,000 set by Biogen.

This has implications for Medicare, and could lead to higher premiums for all 56 million Part B enrollees in traditional Medicare and Medicare Advantage, according to the Kaiser Family Foundation (KFF). As an infusion product, it would be covered under Medicare Part B.

In 2017, nearly 2 million Medicare beneficiaries used one or more of the currently-available Alzheimer’s treatments covered under Part D, based on KFF analysis of Medicare Part D claims data.

KFF speculates that if just one-quarter of these beneficiaries are prescribed Aduhelm and Medicare pays 103% of $56,000 in the near term, total spending for one year alone would be nearly $29 billion. Total Medicare spending for all Part B drugs was $37 billion in 2019.

A July 15 public meeting including Biogen, the Alzheimer’s Association, and the Centers for Medicare and Medicaid Services and other stakeholders will discuss coverage options for Medicare and private payers.