Dissemination, Science

Biogen Aducanumab drug approved by FDA

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The US drug regulator, the FDA, announced that it has granted a licence for the Alzheimer’s drug aducanumab (known commercially as Aduhelm). This means that they view the drug to be safe with some evidence of effectiveness, but have asked the pharmaceutical company Biogen to conduct a further stage four clinical trial to verify the drug’s long-term benefits.

This is a positive step forward for people living with Alzheimer’s in the US, and one that will provide hope for many across the world.

It’s been nearly 20 years since the last drug for Alzheimer’s was made available. Aducanumab, developed by the pharmaceutical company Biogen, is the very first to tackle the disease itself and is for people with mild cognitive impairment and early Alzheimer’s disease.

The news provoked a mixed reaction online and within trusted dementia organisations:

“The FDA’s decision to approve aducanumab marks a hugely significant milestone in the search for much-needed treatments for Alzheimer’s. With no effective therapies currently available to modify the progression of this devastating disease, we all hope it proves a turning point for the millions of people living with the condition.

The Alzheimer’s research field is however polarised on the aducanumab study findings, as some believe that the evidence for clinically meaningful benefits is not sufficient. There are still many barriers to overcome including cost, eligibility and clinicians’ enthusiasm to prescribe a medicine whose effects are far from certain. As we learn more about Alzheimer’s and the best times for treatment intervention, I’m sure we will see more refined therapeutics, likely in combination to target multiple biological components. The trial results appear to indicate that targeting the amyloid beta protein is a viable treatment option and worth pursuing. Indeed, there are other monoclonal antibodies currently in trials and the pharmaceutical company Eli Lilly recently released positive news about their Phase II trial of donanemab.

In the last few years, I have seen encouraging progress in both our understanding of the mechanisms underlying neurodegenerative diseases like Alzheimer’s, and our approach to drug discovery and clinical trials. The search for treatments is difficult as the brain, and disorders associated with it, are extremely complex; there are likely multiple causes involving several biological pathways. Additionally, Alzheimer’s disease progresses over a very long time, with symptoms often only appearing decades after the first biochemical changes in our brain. We are now seeing a shift in clinical trial design whereby we can intervene earlier in the disease, saving large numbers of neurons. This is aided by the development of more precise blood biomarkers that can track stages of progression and predict those at highest risk of developing the disease.

I sincerely hope that the outcome of this decision encourages our colleagues in the pharmaceutical industry. Now more than ever, we need investment in research and drug development for neurodegenerative diseases like Alzheimer’s, which is the most common cause of dementia. Dementia affects nearly 900,000 people in the UK and, with our ageing population, this is projected to rise to 2 million by 2050. In April, dementia overtook Covid-19 as the leading cause of death – a stark reminder of this growing and lasting health crisis. The pandemic has shown us that with sufficient funding and mobilisation of resources, we can make swift and significant progress against disease. If we apply that same urgency to dementia, I’m optimistic we will make further breakthroughs in treatments and improve the lives of those living with the condition.”

Prof Bart De Strooper, Director of the UK Dementia Research Institute

Evidently this is exciting and welcome news; however, it raises many questions in relation to eligibility, access, timescales for individual countries, health system readiness, and of course cost. ADI will endeavour to keep you updated with progress and information.

Chris Lynch, Acting CEO & Director of Policy, Communications & Publications, Alzheimer’s Disease International

Aducanumab is still some way from reaching patients in the UK. Today, Alzheimer’s Research UK has written to the Health Secretary, Matt Hancock, to ask the government to speed up assessment of aducanumab by UK regulators. Read our blog for the latest on aducanumab and our response to this historic ruling.

Aducanumab isn’t suitable for everyone with dementia, but we hope today’s announcement has an immediate positive impact on the global search for effective dementia treatments.

We will continue to keep you updated with the latest developments. Research has the potential to transform the lives of everyone affected by dementia – and thanks to your support, we will keep working to make more breakthroughs possible.

Hilary Evans, Chief Executive

It’s promising to see that aducanumab has been approved for use in people with early stage Alzheimer’s disease – the first drug to be approved in nearly twenty years by the US regulatory authorities. We await the opinion of the European Medicines Agency and the outcome of any application made to the UK regulatory authorities, to give clarity to people with early Alzheimer’s disease in the UK. Whatever the outcome of their decision, this is just the beginning of the road to new treatments for Alzheimer’s disease. As this drug will only benefit a proportion of people in the early stages of Alzheimer’s disease, there are hundreds of thousands more who may not be eligible, so we must keep searching for drugs for all stages of Alzheimer’s disease and for other types of dementia. We are proud to say that Alzheimer’s Society was part of a pivotal genetic discovery for the first Alzheimer’s gene in the 1990s, which paved the way for anti-amyloid drugs like the one announced by Biogen.

‘Research will beat dementia. We have seen tremendous progress over the last decade, leading up to this decision today, but we need to maintain the momentum: the Government must honour their commitment to double dementia research funding. Dementia is the biggest killer in the UK, so we owe it to all 850,000 people living with dementia in the UK and their families to make a world without dementia a reality.

Dr Richard Oakley, Head of Research at Alzheimer’s Society

We, as a community (Researchers), experienced a historic moment today: the approval of a first drug that modifies one of the underlying biological contributors to Alzheimer’s disease. This is the beginning of a completely new future for treatments in Alzheimer’s and other dementia. This new treatment, while not a cure, is significant and we know this will be the first of a number to come. It is important to acknowledge that this FDA-approved treatment slows progression (in a limited population) of MCI due to AD and early Alzheimer’s dementia. It does not stop decline or improve cognition. But, it represents an important step and progress in Alzheimer’s science. We recognize that — as with all drug therapies — there can be heterogeneity in treatment effects. More research will be needed to better understand the benefits, side effects and best application.

The Alzheimer’s Association will do everything in its power to ensure access to the drug and requisite tests for all who will benefit. One of our highest priorities will be eliminating barriers to access.

We know this is just the beginning of more meaningful treatment advances. We must advance effective treatment options for diverse populations living in all stages of Alzheimer’s and other dementia. And we must address Alzheimer’s through multiple pathways — more than just amyloid — with an eye toward effective combination therapies that work at different stages of the disease, pharmacological and nonpharmacological.

I am grateful for all you do every day, and also proud of not only our research and clinical community, but also proud of how the Alzheimer’s Association has contributed significantly to our progress as a partner with all of you.

Thank you for your tireless contributions and the work you will continue to do moving forward. We know this is only the beginning — both for this drug and for better treatments to come for Alzheimer’s and all dementia. It is not time to slow down but to speed up! So many are counting on us, and I know that together we will all work towards the next treatments for these devastating diseases.

Dr. Maria Carrillo, Chief Science Officer, Alzheimer’s Association

As a dementia clinician and researcher with personal family experience of Alzheimer’s disease, I want to see effective dementia treatments as much as anyone. I consider the approval of aducanumab represents a grave error that will have only negative impact on patients and their families & that could derail the ongoing search for meaningful dementia treatments for a decade. The FDA has ignored data from the trial which showed no slowing of decline in cognition or function, that could derail the ongoing search for meaningful dementia treatments for a decade. Amazingly, the FDA have sidestepped available clinical trial outcomes data that indicate the drug probably doesn’t work.

Professor Robert Howard, Old Age Psychiatrist, University College London

There has been considerable public debate on whether Aduhelm should be approved. As is often the case when it comes to interpreting scientific data, the expert community has offered differing perspectives. At the end of the day, we followed our usual course of action when making regulatory decisions in situations where the data are not straightforward, and concluded that the benefits of Aduhelm for patients with Alzheimer’s disease outweighed the risks of the therapy. In all studies in which it was evaluated, however, Aduhelm consistently and very convincingly reduced the level of amyloid plaques in the brain in a dose- and time-dependent fashion. It is expected that the reduction in amyloid plaque will result in a reduction in clinical decline. The FDA will continue to monitor Aduhelm as it reaches the market and ultimately the patient’s bedside.

Dr. Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research

We have to really temper expectations and explain to people that this drug is meant for the earliest symptomatic phases,” he said. “It pains me to say this but if I have a severe Alzheimer’s patient that can no longer speak or interact much with others and their family member is begging me to give them this drug, I won’t be able to do it.

Dr. Richard Isaacson, Director of the Alzheimer’s Prevention Clinic at Weill Cornell Medicine and NewYork-Presbyterian in New York (Lead for one of the trial sites)

The evidence is insufficient to conclude that the clinical benefits of aducanumab outweigh its harms or, indeed, that it reduces progression

Institute for Clinical and Economic Review


Brain Health Scotland has prepared a very balanced set of FAQs which will be helpful looking for more information:

Read the Brain Health Scotland FAQs

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    […] and clinicians’ enthusiasm to prescribe a medicine whose effects are far from certain”, Dementia Researcher […]


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