Biogen has withdrawn its marketing authorization application (MAA) to the European Medicines Agency (EMA) for its Alzheimer’s drug, Aduhelm (aducanumab), a drug that had been targeted for potential blockbuster status. The company’s decision to withdraw its application followed discussions with the EMA’s Committee for Medicinal Products for Human Use (CHMP) that indicated that data provided thus far would not be sufficient to support a positive opinion for marketing authorization of the drug. Biogen’s MAA had been under review by the CHMP in response to the company’s request for a re-examination of a negative opinion issued in December 2021 by the CHMP.

The decision to withdraw the drug for regulatory review in the European Union marks the latest setback for Biogen. The drug was approved under the accelerated approval pathway by the US Food and Drug Administration (FDA) in June 2021, which requires post-approval confirmatory trials to verify clinical benefit. The accelerated approval pathway provides patients with a serious disease earlier access to drugs when there is an expectation of clinical benefit despite some uncertainty about the clinical benefit with follow-up trials required. Earlier this month (April 2022), the US Centers for Medicaid and Medicare Services (CMS) released a national policy for coverage under Medicare, the US federal healthcare program for individuals 65 or older, to restrict Medicare coverage for anti-beta-amyloid drugs for treating Alzheimer’s disease, such as Aduhelm, only to patients taking part in approved clinical trials, which includes post-approval confirmatory trials as part of FDA’s accelerated approval pathway.

The FDA’s approval of Aduhelm was the first approval in almost two decades of a new treatment for Alzheimer’s disease but its approval came with some issues. Prior to getting the accelerated approval by the FDA, an FDA advisory committee had earlier recommended not to approve the drug, citing the need for additional research. Recommendations by FDA advisory committees are non-binding, but are usually in line with final FDA decisions, although not in this particular case.

Aduhelm was slated for blockbuster status by some analysts, but thus far the drug has performed below expectations. Following accelerated approval in June 2021, Biogen posted full-year 2021 sales of $3 million.

Source: Biogen