On the 7th of June 2021, the United States Food and Drug Administration (FDA) approved the application to license a new drug - aducanumab – for the treatment of Alzheimer’s disease. Aducanumab also gets referred to by its brand name - Aduhelm.
This drug has received a lot of media attention for two main reasons. Firstly, despite lots and lots of clinical trials, there hasn’t been a new drug approved for Alzheimer’s disease since 2003. Secondly, all the drugs we currently have for Alzheimer’s disease are symptomatic treatments; they act to help ease symptoms, but they don’t do anything to stop or slow down the disease process that causes these symptoms. Aducanumab may be the first disease modifying therapy - a drug that acts to slow the progression of disease.
Imagine the damage caused by Alzheimer’s disease in the brain is equivalent to a boat springing a leak. The drugs we currently have act to bail water out of the boat, but they don’t do anything to plug the hole. Plugging the hole is the aim for disease modifying therapies.
The aducanumab journey has been a bit of a rollercoaster over recent years and its application for licensing approval has stirred a lot of debate.
Dr Terry Quinn explains what we know so far
Read our FAQs below to find out more. If you have suggestions for any more questions you would like to see answered get in touch.
UPDATE 17th December 2021: The European Medicines Agency (EMA) - the regulator who decides if a new drug gets approved for use in the European Union - have announced their decision to refuse a license for aducanumab for the treatment of Alzheimer's disease.
The EMA is the equivalent of the FDA in America - who decided to give accelerated approval to the drug back in June 2021 (see ‘What does accelerated approval and phase IV mean for aducanumab’ below).
So this latest announcement means that aducanumab will not be approved for clinical use in the EU at this moment in time but may well become available through future research programmes.
The decision on whether to approve aducanumab for use in the UK is not down to the EMA and instead sits with a separate UK-specific regulator – the MHRA. We haven’t heard the MHRA’s verdict on aducanumab yet but this decision not to license the drug in Europe makes it very likely the drug will also not be approved at this time in the UK.
The process of applying for a licence towards a new medication is dynamic. Decisions are re-visited as new evidence emerges from clinical trials and other research. The manufacturers of aducanumab are collecting new data all the time and these data may strengthen the case in support of the medication.
For more on the regulatory approval process see our section below ‘Who are the FDA, MHRA and EMA and what is their role?’
UPDATE 11th January 2022: The Centers for Medicare and Medicaid Services (the government health insurance provider in the US) have announced a draft decision to only cover the cost of aducanumab for people who are enrolled in approved clinical trials. This essentially means that the only people who will have access to aducanumab at this moment in time are those taking part in a research study designed to further investigate the safety and effectiveness of the drug.
This provisional decision by the US providers (to be finalised by 11th April) also extends to other potential new drugs in development that aim to work in a similar way to aducanumab – by breaking down the clumps of amyloid protein in the brain.
Brain Health Scotland works closely with Biogen to advance the brain health environment in Scotland and has received funding from Biogen to carry out research projects.
These FAQs were written by Dr Catherine Pennington, Dr Terry Quinn, Prof Craig Ritchie and Dr Jenny Waymont.
Dr Catherine Pennington is currently funded by the Clinician Scientist Office in Scotland. She previously worked on the EPAD study, and received grant funding from the BRACE charity, and training funded by Merz Pharmaceuticals. She is also an NHS consultant neurologist working for NHS Forth Valley and NHS Lothian.
Dr Terry Quinn has received honoraria, to his University, for his participation as a member of an advisory board for Novartis and as chair of a Data Monitoring Committee for Novo-Nordisk. Terry’s University has received speaker fees for non-promotional educational symposia organised by Bayer, BMS and Pfizer. Terry’s University has received investigator initiated research funding for studies supported by BMS-Pfizer Alliance where he is the Principal Investigator.
Prof Craig Ritchie has been a paid consultant for several companies developing treatments for Alzheimer’s disease over the last 5 years including Biogen, Eli Lilly, Merck, Roche, Janssen, Abbvie, Kyowa Kirin, Actinogen and Eisai. He was the UK Chief Investigator for the ENGAGE Trial and Academic Lead on the EPAD (European Prevention of Alzheimer’s Dementia) Programme which was a public:private partnership between the EU and several companies with an interest in developing treatments for AD www.ep-ad.org His unit at the University of Edinburgh (Edinburgh Dementia Prevention) has received grant funding from Biogen, Janssen, AC Immune and Actinogen. He is the unpaid chairperson of the Brain Health Clinic Consortium established in the UK by Biogen.
Dr Jenny Waymont is currently funded through an educational grant provided by Biogen (who developed aducanumab), and has previously received funding from TauRx Pharmaceuticals (who develop drugs targeting Tau). Jenny holds a PhD in Medical Imaging, but is not a medical doctor.