The Food and Drug Administration (FDA) has granted accelerated approval for the monoclonal antibody, aducanumab (Aduhelm), a first-in-class drug, for treatment for Alzheimer’s disease.
Alzheimer’s disease can be described as an irreversible, progressive brain disorder that slowly destroys memory and thinking skills and ultimately, the ability to carry out simple tasks. The precise cause of the disease is still not fully clear but a defining feature in the brain of sufferers is an accumulation of amyloid beta plaques and neurofibrillary, or tau, tangles which result in loss of neurons and their connections. Aducanumab works by targeting the aggregated soluble and insoluble amyloid beta plaques.
The efficacy of Aduhelm has been evaluated in three separate clinical studies with a total of 3078 patients and which have been described by the manufacturer, Biogen, in their prescribing information leaflet. The dosage is 10 mg/kg and the drug is administered over one hour every 4 weeks and available at two different strengths, 170mg and 300mg.
In the clinical trials, the effect of Aduhelm on amyloid plaques was assessed using positron emission tomography (PET) and the results showed a significant reductions in plaques after 26 and 78 weeks of treatment. Nevertheless, writing on the FDA site, Dr Patrizia Cavazzoni, Director, FDA Center for Drug Evaluation and Research noted that “the data included in the applicant’s submission were highly complex and left residual uncertainties regarding clinical benefit.” However, she added that “the Agency concluded that the benefits of Aduhelm for patients with Alzheimer’s disease outweighed the risks of the therapy.”
The FDA has stipulated that Biogen has to conduct a post-approval clinical trial to verify the drug’s clinical benefit. If the drug does not work as intended, then the FDA can take steps to remove it from the market.