The US FDA has recently approved a new Alzheimer’s disease drug Aducanumab to be sold under the brand name Aduhelm (Biogen). It is the first new treatment approved for Alzheimer’s since 2003 and is the first therapy that targets the fundamental pathophysiology of the disease.
Aducanumab is an amyloid beta-directed antibody indicated for the treatment of AD. Researchers evaluated the drug’s efficacy in three separate studies representing a total of 3,482 patients. The researchers demonstrated that patients receiving the treatment had a significant dose-and time-dependent reduction of amyloid-beta plaque, while patients in the control arm of the studies had no reduction of amyloid beta plaque. Amyloid beta plaque was quantified using positron emission tomography (PET) imaging to estimate the brain levels of amyloid beta plaque in a composite of brain regions expected to be widely affected by AD pathology.
The drug includes a warning for amyloid-related imaging abnormalities (ARIA), which most commonly presents as temporary swelling in areas of the brain that usually resolves over time. Some people may have symptoms such as headache, confusion, dizziness, vision changes, or nausea. Another warning for aducanumab includes a risk of hypersensitivity reactions, including angioedema and urticaria. The most common side effects of the drug were ARIA, headache, fall, diarrhoea, and confusion/delirium/altered mental status/disorientation.
Aducanumab is currently approved under the accelerated approval pathway, which provides patients suffering from a serious disease with earlier access to drugs when there is an expectation of clinical benefit despite some uncertainty about the clinical benefit of the drug, revealed the agency. 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.
“Currently available therapies (for AD) only treat symptoms of the disease; this treatment option is the first therapy to target and affect the underlying disease process of Alzheimer’s. As we have learned from the fight against cancer, the accelerated approval pathway can bring therapies to patients faster while spurring more research and innovation,” said Patrizia Cavazzoni, M.D., director of the FDA’s Center for Drug Evaluation and Research.