FDA grants aducanumab approval for Alzheimer’s disease

10th June 2021

The US Food and Drug Administration (FDA) has granted accelerated approval for the monoclonal antibody, aducanumab, a first-in-class drug, for treatment for Alzheimer’s disease.

Alzheimer’s disease is 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 (Aduhelm) works by targeting the aggregated soluble and insoluble amyloid beta plaques.

The efficacy of Aduhelm has been evaluated in three separate studies with a total of 3078 patients and which have been described in the manufacturer’s prescribing information leaflet. The dosage is 10mg/kg and the drug is administered over one hour every 4 weeks and available at two different strengths, 170 mg and 300mg.

In clinical studies, the effect of Aduhelm on amyloid plaques was assessed in the trials using positron emission tomography (PET) and resulted in significant reductions in plaques after 26 and 78 weeks of treatment. 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.” Nonetheless, she also added that “the Agency concluded that the benefits of Aduhelm for patients with Alzheimer’s disease outweighed the risks of the therapy.”

Additionally, the FDA is requiring the manufacturer, Biogen, 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.

FDA approves remdesivir for COVID-19

30th October 2020

The US Food and Drug Administration (FDA) approved remdesivir (brand name Veklury) on 22 October 2020 for use in adults and paediatric patients aged 12 years and over and weighing at least 40kg for the treatment of COVID-19 in patients requiring hospitalisation.

The approval requires that remdesivir is only used in a hospital or healthcare setting capable or providing acute care which is comparable to hospital care. This new approval does not however, include the entire population that was originally included via the emergency use authorisation (EUA) issued on 1 May 2020. In order to allow continued use in paediatric patients the EUA was amended for use only in laboratory confirmed COVID-19 cases for patients weighing 3.5 to less than 40kg less or hospitalised children under 12 years of age weighing at least 3.5kg. However, the FDA makes clear that this is NOT an approved use of the drug and that this authorisation is only temporary and could be revoked.

The approval in adults was based on three randomised clinical trials which showed that treatment with remdesivir lead to clinically meaningful improvements across multiple outcomes compared to placebo. For instance, in the most recent ACTT-1 trial, published in the New England Journal of Medicine, remdesivir significantly improved time to recovery by 5 days and reduced disease progression among patients requiring oxygen. It also showed an improved time to recovery among patients not requiring oxygen in the SIMPLE-Moderate trial conducted in hospitalised patients. Moreover, adverse effects with remdesivir were similar to placebo.
Remdesivir was approved under the early access to medicines scheme in the UK in May 2020.

Reference
FDA News release. https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-covid-19