US regulators recently approved Biogen’s aducanumab drug as a treatment to address an underlying cause of Alzheimer's disease.
The drug is not currently available for use in the UK, but could the Food and Drug Administration’s (FDA) recent decision in the US prompt approval of aducanumab on the NHS?
What is aducanumab?
Aducanumab is an antibody which is designed to remove amyloid plaque from the brain, which forms when protein pieces called beta-amyloid clump together.
Beta-amyloid comes from a larger protein found in the fatty membrane surrounding nerve cells and can gradually build up into plaques.
Professor Craig Ritchie, Director of Brain Health Scotland and Professor of the Psychiatry of Ageing, University of Edinburgh, said that although the FDA’s approval of aducanumab “is welcomed as it opens a new chapter for drug treatments in Alzheimer’s disease”, it doesn’t mean it will be immediately approved in the UK.
Prof Ritchie added: “Use within the NHS though remains some months away and it is critically important that the safe use of this intervention in those people most likely to benefit is fully considered.
“We also need to ensure that we collect real world data on the benefits and side effects of the medication to help us to refine how to use this intervention well. It’s certainly a new chapter but by no means the end of the story.”
The FDA’s approval of the drug was controversial, due to only one of two large-scale trials showing that aducanumab significantly slowed progression of Alzheimer’s.
However, Pamela Spicer, Principal Analyst at Informa Pharma Intelligence, said that the FDA’s decision was “a groundbreaking moment in the research and treatment of Alzheimer’s disease”.
She added: “Not only has the world had to wait almost 20 years for a new Alzheimer’s drug, but this is also thought to be the first ever approved treatment designed to slow the progression of the disease in people with early stage cognitive impairment, rather than just masking the symptoms.”
Despite this, Ms Spicer does note that the FDA’s approval was “unusual” as “it goes against the prior recommendation of an advisory panel, whose judgement was that the current data were insufficient”.
When could aducanumab be available to patients in the UK?
Addressing if aducanumab could soon be available for use in the UK, Ms Spicer explains that Biogen has not yet filed aducanumab in the UK, which is “a necessary first step before anything else can happen”.
Approval decisions in the UK are then typically expected within a year of filing.
However, Ms Spicer explains that reimbursement costs “will be a major hurdle in markets like the UK”, especially as questions also remain surrounding how well the biomarkers that the FDA used - amyloid plaque reduction - correspond to cognitive benefit.
Ms Spicer notes that the UK’s cost-effectiveness regulator, NICE, “is one of the strictest bodies globally”.
In the US, aducanumab is set to cost tens of thousands of dollars annually per patient.
Alongside this, Ms Spicer says it’s important to take into account the delivery of the drug and facilities needed to administer repeated infusions in elderly patients.
“In some areas, mental health services may be community-based and not necessarily set up basically to administer biologics.
“Based on this, realistically, we are still some way from UK patients potentially benefiting from the drug,” Ms Spicer adds.