The US Food and Drug Administration (FDA) has approved Aduhelm (aducanumab) to treat patients with Alzheimer’s disease using the accelerated approval pathway.
Aduhelm is the first treatment directed at the underlying pathophysiology of Alzheimer’s disease, the presence of amyloid beta plaques in the brain.  The clinical trials for Aduhelm were the first to show that a reduction in these plaques—a hallmark finding in the brain of patients with Alzheimer’s—is expected to lead to a reduction in the clinical decline of this devastating form of dementia.
The late-stage development program for Aduhelm consisted of two phase III clinical trials. One study met the primary endpoint, showing reduction in clinical decline. The second trial did not meet the primary endpoint.  In all studies in which it was evaluated, however, Aduhelm consistently 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.
Michel Vounatsos, Chief Executive Officer at Biogen said: “For nearly two decades, the medical and scientific communities have searched for a new treatment for Alzheimer’s disease. And millions of people with this dreaded condition—along with tens of millions more of their family, friends and caregivers—have watched helplessly, with little hope, as they confronted this devastating condition. In my own experience, I too have seen how this disease robs patients of their cognitive abilities, of their capacity to enjoy their lives and interact with loved ones.
“The approval of Aduhelm represents a crucial inflection point in our collective battle against Alzheimer’s disease. By addressing a defining pathology of the disease, this novel therapy has the potential to help fundamentally change the way patients are diagnosed and treated.
“I have hoped for years that we would reach a moment like this. We all know the staggering numbers: there have been at least 100 drug development programs discontinued since 2003—the last time a new Alzheimer’s drug was approved. What it tells us is that the path for innovation is not straightforward, especially for something as complex as Alzheimer’s research. The journey during Biogen’s many years of research and development has been humbling, but we have learned from industry’s past research efforts and been determined to follow the science, always driven to address patients’ unmet needs.
“Aduhelm is a first-in-class approved therapy: I believe it will be the catalyst to a new era of innovation for Alzheimer’s disease and the first of many new treatments available to patients. More resources will be drawn into research that can help patients through the disease continuum, explore new pathways, and find potential therapy combinations.”
Industry comment
Dr Gen Li, Founder and President of virtual clinical development company Phesi said: “Beyond the immediate implications for Biogen, what this debate around aducanumab has underscored is that neurodegenerative diseases like Alzheimer’s remain an incredibly emotive and fraught subject. For Biogen, it might have been a “bet the farm” approach, but for sufferers, their families and their carers, even the slightest possibility of a treatment naturally holds much hope. The landmark FDA decision made today will be discussed at length, but it’s important to remember that neurodegenerative disease is one of the biggest unmet needs humanity faces and continued R&D is imperative. The WHO estimates that 50 million people around the world suffer with dementia, with Alzheimer’s contributing up to 70% of those cases. 10 million new cases are added every year.
Though the life sciences industry has greatly improved its understanding of Alzheimer’s, we are yet to capitalize fully on what we have learned. Advances in AI and data science mean we can gain an unprecedented level of detail on the disease from existing data. Our knowledge of genomics means we have a better understanding of how therapeutics can work for individual patients. And real-world patient data will be a critical component. But without the inclination and ability to bring these data sources together, insights will remain hidden. Regulators, companies, governments and patient advocacy groups have the same end goal in mind. Working alongside regulators is critical, as is ensuring the integrity of trials. We believe that by working together, and sharing data and expertise, further breakthroughs will be made.”