A new treatment for Alzheimer’s disease was approved by the US Food and Drug Administration on 7 June using a surrogate end point for clinical benefit: a reduction of amyloid beta plaque in the brain. Aduhelm (aducanumab) was given an accelerated approval. It is the first new treatment approved for Alzheimer's disease in the US since 2003.
The approval comes two years after the developers, Biogen Inc and Eisai Co Ltd, stopped two Phase 3 trials of aducanumab for lack of efficacy. They restarted the programme after analysing additional data that suggested a beneficial effect.
“Currently available therapies only treat symptoms of the disease; this treatment option is the first therapy to target and affect the underlying disease process of Alzheimer’s,” said Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research.
Aduhelm is a monoclonal antibody that binds to amyloid beta plaque in the brain with the goal of clearing it. While the specific causes of Alzheimer’s disease are not fully known, a leading hypothesis is that the disease is a result of a build-up of amyloid plaques and neurofibrillary, or tau, tangles in the brain. This results in a loss of neurons and their connections, progressively destroying memory and thinking skills. Aduhelm was tested in three separate placebo-controlled trials of 3,482 patients with the disease. Patients receiving the drug had a significant dose-and time-dependent reduction of amyloid beta plaque while those in the control arm had no reduction.
Amyloid beta plaque was quantified using positron emission tomography imaging to estimate the brain levels of amyloid beta in a composite of brain regions expected to be affected by Alzheimer’s disease pathology compared with a brain region expected to be spared of such pathology, the FDA said. Under the accelerated approval, the companies will need to conduct another trial to verify the drug’s expected benefit.
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