Aduhelm is an amyloid beta-directed antibody indicated for the treatment of Alzheimer’s disease. It is approved under the accelerated approval pathway, which provides patients suffering from a serious disease earlier access to drugs when there is an expectation of clinical benefit despite some uncertainty about the clinical benefit of the drug.
Accelerated approval is based upon the drug’s effect on what is called 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.
This pathway requires the company to verify clinical benefit in a post-approval trial. If the sponsor cannot verify clinical benefit, the FDA may initiate proceedings to withdraw approval of the drug.
- Press Release
- From Our Perspective
- Letter to Advisory Committee
- November 6, 2020: Meeting of the Peripheral and Central Nervous System Drugs Advisory Committee Meeting Announcement