Following the news that the FDA had approved aducanumab for the treatment of Alzheimer’s disease, the European Alzheimer’s Disease Consortium (EADC) published a position statement, on 15 June 2021.
It begins by noting that the FDA approval is subject to the drug's manufacturer, Biogen, conducting an additional clinical trial at Phase IV, to verify the drug's clinical benefit, and that if the study were to fail, the approval may be revoked. The EADC also highlights that the drug is currently only approved in the US, and emphasises that aducanumab trials only enrolled a group of patients with a narrow disease–specific stage, namely mild cognitive impairment (MCI) due to Alzheimer's disease (AD) or early-stage Alzheimer's dementia, and that aducanumab should therefore only be given to this group of patients.
It also stresses that, given that the FDA followed a different regulatory pathway, i.e. that of a surrogate endpoint (approval on the basis of the evidence of reduction of amyloid deposits), the approval may open the door for many other applications based on amyloid reduction, but without solid evidence for clinical efficacy. Due to the absence of convincing clinical benefits, this is and will remain controversial, pending further evidence. Indeed, the EADC points out that further research is needed to better understand which patients and at what disease stage they respond best, what is the optimal duration of treatment (current data do not span more than 18 months) and what side effects may occur after long-term use.
In conclusion, the statement says that, while doctors and patients have been hoping for a new and improved drug for AD, for many years now, caution is advisable in view not only of the high cost and of the expectations surrounding the new drug, but also of the uncertainty around the lack of clear clinical proof of efficacy, at this stage. On a more positive note, the EADC points out that despite these uncertainties, this first treatment against the pathophysiology of AD is of great importance and if eventually proven to slow the progression of cognitive decline in people in the MCI stage of AD or early Alzheimer's dementia, will be a major advance in the field. It also expresses the hope that the use of aducanumab will represent a breakthrough for more AD therapies and encourage more pharmaceutical companies to resume their research activities, including into targets other than amyloid.
Read the EADC’s full position statement, here:
http://www.eadc.info/public/documents/external_documents/Statement-Approval-Aducanumab-EADC.pdf