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The U.S. Food and Drug Administration (FDA) has converted lecanemab from an accelerated approval to a traditional approval for the treatment of Alzheimer’s disease.
Prof Tara Spires-Jones, President of the British Neuroscience Association, Group leader at the UK Dementia Research Institute, and Deputy Director of the Centre for Discovery Brain Sciences at the University of Edinburgh, said:
“The news that lecanemab has been approved to treat Alzheimer’s disease in the USA is very welcome. This is the first approved therapy that slows the progress of disease. The treatment works by directly attacking one of the pathological proteins that clumps in the brain, but it is not a cure and only modestly slows cognitive decline. There are also serious side effects in some people. While not perfect, this approval provides hope for people living with dementia and motivation for scientists and funders who now know in principle that the disease can be stopped.”
Dr Adrian Ivinson, Director (Operations), COO, UK Dementia Research Institute (UK DRI), said:
“This further FDA approval of Lecanemab is both expected and welcome. Any disease-modifying drug with good evidence that it helps some patients is an important step forward.
“However, Lecenemab is not a panacea. The reported effect on patients is real but modest. It doesn’t offer a cure—it won’t repair the damaged brain or even halt the disease process. What it will do, for at least some patients, is slow the rate of decline. It is only recommended for patients with early disease—a minority of the AD population—and there are rare but important side effects to be monitored and manged. It is also an expensive drug, both in terms of the cost of the drug itself and the procedures required to identify and treat patients. All this means that it is unlikely to be widely available in the UK for some time.
“It is nonetheless very good news. Why? Because for the first time it shows that drugs that can alter the Alzheimer’s disease course are possible. This will bring companies and investors back to the table. It also provides a massive shot in the arm for the not-for-profit research community. For decades we have been telling anyone who will listen that there is light at the end of the tunnel. Today that light is just a bit brighter and a bit closer. And it provides hope to everyone with or at risk of AD.
“What is needed now is a redoubling of our collective efforts. As a society we must dramatically increase public funding for AD research including the basic discovery science that made Lecanemab possible and the translational research and infrastructure building that is needed to deliver the next generation of drugs to all AD patients.”
Dr Tom Russ, Director of the Alzheimer Scotland Dementia Research Centre, and Reader in Old Age Psychiatry, University of Edinburgh, said:
“The news that Lecanemab has been fully licensed by the FDA in the USA will bring hope to many people living with dementia and their families. However, the fact remains that the effect of this drug is modest and associated with potentially serious side effects. Furthermore, it seemed to work less in women than men, if at all. However, while this is another encouraging development, the UK NHS is not ready to implement an infusion-based therapy, should it become licensed here. There needs to be significant investment in and support for struggling dementia assessment services which are still emerging from the Covid-19 pandemic to continue to diagnose, treat, and support people currently presenting while developing new ways to implement the disease-modifying treatments of the (near) future.”
Dr Ivan Koychev, Senior Clinical Researcher, Dementia Platform UK, University of Oxford, said:
“The full approval of lecanamab signals a step change in the treatment of Alzheimer’s disease. It clears the amyloid proteins that are part of the Alzheimer’s process and the approval rests on data from studies showing some modest decrease in the rate at which patients decline. Concerns remain about the safety and deliverability of these therapies; in the UK for example the drug would require a complete overhaul of the memory clinics to allow intravenous drug administration, genetic testing to determine those most likely to experience side effects and serial MRI head scanning to monitor for side effects. While these issues will undoubtedly limit the access to the drug itself, this new frontier in the treatment of Alzheimer’s disease will stimulate investment in the area to further refine treatments that change the disease process and will make the case for investment in non-pharmacological programmes for prevention of the disease in people at risk.”
Dr Nick Fox, Director of the Dementia Research Centre and Professor of Clinical Neurology, UCL, said:
“This is milestone in the search for effective therapies for Alzheimer’s disease – this, the first full approval by the FDA, supports the widely held view in the field that this drug does deliver clinical benefit and slows disease progression. This should accelerate approvals globally and focus minds on the challenge of delivering therapies effectively and equitably in stretched and unprepared health care systems. It adds to a growing sense of hope that finally we are making progress against this devastating disease.”
Prof James Rowe, Professor of Cognitive Neurology in the Department of Clinical Neurosciences at the University of Cambridge, and Assistant Director of Dementias Platform UK, said:
“This is a very important decision by the FDA. It highlights the need for the UK to plan now to be able to deliver disease-modifying treatments for people Alzheimer’s disease. There is much to be done if lecanemab and similar drugs are to be accessible with safety and equity – better diagnosis, genotyping and issues that raises, improved MRI access, and the specialist NHS staff and sites to safely administer advanced therapies.
“In the UK, we need MHRA and NICE to complete their reviews on lecanemab with the urgency that is felt by so many families. The FDA has not made the decision lightly. The evidence on lecanemab includes practical information on caveats and risks, to help clinicians and patients make individual decisions.
“This FDA decision will increase the accessibility of lecanemab in the US. There is still much to be learnt about the drug and its long term effects. But one thing we have all learnt is that investment in dementia research delivers results.”
Dr Mark Hibberd, Chief Medical Officer, Pharmaceutical Diagnostics, GE HealthCare, said:
“This is an exciting development for patients and caregivers, though limitations to access the treatment remain. Amyloid-targeted therapies like this require confirmation of the presence of amyloid beta pathology before being administered, but access to positron emission tomography (PET) amyloid imaging – the only method to directly visualize amyloid brain depositions in patients – is limited, primarily due to lack of reimbursement in the U.S., but also in other geographies, including the UK. We are optimistic that coverage for amyloid PET scans will follow as disease modifying therapies are more widely adopted.”
Laurence Barker, partner at the Dementia Discovery Fund, said:
“The clinical data and subsequent approval of Lecanemab shows that Alzheimer’s disease is modifiable, which is great news for patients. This mechanism is the first to be approved but surely won’t be the last. There are over 100 additional agents currently being developed for Alzheimer’s. This is just the beginning of tackling all aspects of the disease.”
Prof Robert Howard, Professor of Old Age Psychiatry, UCL Division of Psychiatry, UCL, said:
“The benefits of lecanemab are so modest as to be undetectable in an individual treated patient. Although 27% slowing of disease course sounds impressive, this is not strictly what the analysis of the trial data showed. It’s important the results are discussed honestly, accurately and without spin. Importantly and unusually, the FDA have issued a Black Box warning alongside the approval because of the incidence of brain swelling and bleeding seen with the drug. Physicians, patients and families will need to weigh up the risks of serious damage and even death against tiny potential advantages of treatment. A small number of people with very mild Alzheimer’s disease will have their condition seriously worsened or may die as a consequence of treatment. I’m afraid I would not want to give this to one of my patients or to a member of my family who had Alzheimer’s disease.”
Prof Bart De Strooper, Professor of Alzheimer’s Disease at UK-DRI (the UK Dementia Research Institute), UCL, said:
“This is a major step forward for the field and a triumph for basic research that has provided the basis for this breakthrough.
“It is however clear that this can only be the beginning. We need cheap and more effective treatment and prevention for all neurodegenerative diseases.
“The approval of Lecanemab provides the proof that this is possible.”
Dr Richard Oakley, Associate Director of Research and Innovation, Alzheimer’s Society, said:
“We’ve waited 20 years for new drugs to treat Alzheimer’s disease, so it’s incredibly encouraging to hear that lecanemab has been approved in the US, particularly as Alzheimer’s Society funded research 30 years ago paved the way to its development.
“However, there is still a long way to go before we see lecanemab available on the NHS. Regulatory bodies in the UK will still need to be satisfied that it’s safe, effective, and cost-effective too.
“It’s also important to remember lecanemab is only effective for people with early Alzheimer’s disease. This means thousands of people with other types of dementia wouldn’t be eligible, and the sad fact is that the majority of people living with Alzheimer’s disease in the UK are not diagnosed early enough to be eligible for early stage treatments such as lecanemab.
“We can’t end up in a situation where there are new drugs being approved but people in the UK can’t benefit. This is why Alzheimer’s Society will keep on calling for more accurate and timely dementia diagnoses, and for our health system to be ready to deliver breakthrough treatments when they come.”
Prof Paresh Malhotra, Head of the Division of Neurology, Imperial College London; and Consultant Neurologist, Imperial College Healthcare NHS Trust, said:
“The approval of Leqembi by the United States FDA is a major step. The ‘black box’ warning indicates the importance and potential severity of the side effects of the drug, which are linked to patients’ genetics and also whether they are taking blood thinners. The informed consent process will be critical to the prescription and delivery of Leqembi, and genetic testing will be part of that. All this will raise a number of issues for what may happen in the NHS, and if Leqembi is approved here, then services will need to be very carefully designed to provide assessment, careful and honest informed consent, regular infusions and monitoring scans, as well as dealing with the side effects of brain swelling and bleeding.”
Prof Sir John Hardy, Professor of Neuroscience and Group Leader at the UK Dementia Research Institute, UCL, said:
“This is clearly great news and heralds a sea change in our views about Alzheimer’s disease: there are now drugs which all agree slow the underlying disease process. This, from Eisai, is the first, and it is likely that the equivalent drug from Lilly is close behind. Safe administration of these drugs to the appropriate patient group will pose an organisational challenge to the NHS, but this is wonderful news. It is worth noting that the UK Alzheimer charities funded the original work leading to this breakthrough and are now funding initiatives to help prepare for appropriate patient identification.”
Prof David Curtis, Honorary Professor, UCL Genetics Institute, UCL, said:
“This really is remarkable news. Certainly there are concerns about serious side effects and questions about the balance between clinical benefits and the overall costs of treatment. Nevertheless, this decision badges Alzheimer’s disease as an illness which can potentially be treated or prevented, rather than simply endured.”
Hilary Evans, Chief Executive at Alzheimer’s Research UK, said:
“This welcome news from the US signals a step towards a world where Alzheimer’s disease is treatable, but should also serve as a wake-up call.
“Today’s ruling involved careful review of data from a rigorous study of 1,800 patients. A similar process is currently underway in the UK, led by the Medicines and Healthcare products Regulatory Agency (MHRA). We’ve written to the MHRA urging them to complete this review as a matter of urgency, without compromising on quality, so people with Alzheimer’s in the UK aren’t left in limbo. We believe that a rapid regulatory decision on lecanemab will further strengthen the UK’s international reputation as a leader in dementia, attract even greater commercial investment in UK clinical trials, and ultimately benefit the one in two of us who will either develop dementia in the future, or care for someone who has it.
“With a UK decision on the horizon, today’s news underlines the urgency of getting the NHS ready for new medicines like lecanemab. There are several barriers that need to be tackled, starting with an overhaul of the way dementia is diagnosed. People won’t be able to access these drugs without a confirmed diagnosis of Alzheimer’s, but this is primarily done using a PET scan and the UK has one of the lowest numbers of scanners per capita in the developed world. We’re concerned that, without action, this would significantly restrict access and create inequities in care.
“Looking further ahead, new diagnostics are on the horizon – blood tests for Alzheimer’s have been developed, and we’re supporting research to hasten their use in clinical practice. So lecanemab’s potential arrival should act as a warning to accelerate work on this front, and help ensure people who could benefit from these new medicines are identified quickly.
“Progress for people with Alzheimer’s disease is long overdue. As a first-generation treatment, lecanemab’s effects are modest – a 27% slowing of progression over 18 months – and it comes with side effects that can be serious. Science can, and must, do better – both in terms of delivering drugs that are even more effective, with fewer side effects, and also treatments for people with later stage Alzheimer’s and those with other forms of dementia. At Alzheimer’s Research UK, we will work tirelessly to help bring about a world free from the heartbreak of dementia, and for a cure for the diseases that cause it.”
Declared interests
Prof Tara Spires-Jones: “I have no conflicts of interest.”
Dr Adrian Ivinson: “No interests to declare.”
Dr Tom Russ: “Tom Russ is a Reader in Old Age Psychiatry at the University of Edinburgh. He is Director of the Alzheimer Scotland Dementia Research Centre at the University of Edinburgh, an honorary Consultant Psychiatrist in NHS Lothian, and Clinical Research Champion of the NHS Research Scotland (NRS) Neuroprogressive & Dementia Network. He is a PI on the Evoke and Evoke+ trials but has no financial involvement with any pharmaceutical company.”
Dr Ivan Koychev: “Ivan Koychev is a member of a NICE Technology Assessment Committe and Chief Medical Officer for Five Lives SAS, a digital healthcare company focusing on prevention of dementia.”
Dr Nick Fox: “I have served on advisory boards for Roche/Genentech, Biogen, Lilly, Ionis, and Siemens – all fees paid to UCL; I served on a Data Safety Monitoring Board for Biogen.”
Prof James Rowe: “James is Professor of Cognitive Neurology in the Department of Clinical Neurosciences at the University of Cambridge, and Assistant Director of Dementias Platform UK. He is also Director of the Cambridge Centre for Frontotemporal Dementia and Related Disorders. At DPUK he leads the Experimental Medicine Incubator (EMI) and the Synaptic Health theme within the EMI.”
Dr Mark Hibberd: “GE HealthCare offers a range of products and solutions to support the Alzheimer’s care area across diagnosis and monitoring, including an FDA-approved radiopharmaceutical that can detect, quantify and visualize beta amyloid in the brain.”
Laurence Barker: “The Dementia Discovery Fund invests in companies pursuing a diverse range of novel approaches to treating the disease. We have Lilly as an investor in our funds and Eisai is an investor in DDF companies.”
Prof Robert Howard: “No conflicts of interest.”
Prof Bart De Strooper: “No direct profits from this drug. But consultant for several pharma and biotech including Eisai.”
Dr Richard Oakley: “Nothing to declare.”
Prof Paresh Malhotra: “I am lead a NIHR-funded Clinical Trial of a symptomatic treatment in Alzheimer’s Disease for which drug and placebo are provided by Takeda (formerly Shire) pharmaceuticals.
I am Deputy Chair of the Alzheimer’s Society Research Council.
I have received a speaker honorarium from GE.
I carry out clinical work at Imperial College Healthcare NHS Trust and Cleveland Clinic London.”
Prof Sir John Hardy: “I have consulted for Eisai and Lilly.”
Prof David Curtis: “I declare no conflict of interest.”
For all other experts, no reply to our request for DOIs was received.