Aducanumab (marketed as Aduhelm) Information
Aduhelm is an amyloid beta-directed antibody indicated to treat Alzheimer’s disease. Aduhelm is approved under the accelerated approval pathway, which 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.
Accelerated approval is based upon the drug’s effect on a surrogate endpoint — an endpoint that reflects the effect of the drug on an important aspect of the disease — where the drug’s effect on the surrogate endpoint is expected, but not established, to predict clinical benefit. In the case of Aduhelm, the surrogate endpoint is the reduction of amyloid beta plaque. The accelerated approval pathway requires the company to verify clinical benefit in a post-approval trial. If the sponsor cannot verify clinical benefit, FDA may initiate proceedings to withdraw approval of the drug.
FDA has posted CDER’s Office of Neurology’s Summary Review Memorandum describing the agency’s extensive review of the Aduhelm application and the basis for approval. FDA also posted the Concurrence Memorandum by Peter Stein, MD, director of CDER’s Office of New Drugs (of which the Office of Neurology is a part), and the Concurrence Memorandum by Patrizia Cavazzoni, MD, director of CDER. The remaining scientific review documents in the Aduhelm action package are not yet available, but will be made available to the public as soon as the internal process of review and redaction is complete.