Michel Vounatos, CEO, Biogen, said he was ‘incredibly humbled’ to share that the therapy had been granted accelerated approval.
The monoclonal antibody is the first new treatment approved for Alzheimer’s since 2003. Unlike currently available therapies – which only treat symptoms of the disease – Aduhelm is the first therapy to target and affect the underlying disease process of Alzheimer’s.
Alzheimer's research: a 99.6% failure rate
Alzheimer’s is an irreversible, progressive brain disorder that slowly destroys memory and thinking skills, and eventually, the ability to carry out simple tasks.
The diseases affects an estimated 6.2 million Americans (and an estimated 35 million people worldwide), and the impact is only expected to worsen with an aging population.
And yet despite the pressing need, developing treatments has proved to be a difficult task. The last time a drug for Alzheimer’s was approved by the FDA was in 2003. Since then, however, there have been at least 100 drug development programs discontinued: demonstrating the complexities of Alzheimer’s research. In fact, during the period 2002-2012, the 413 Alzheimer’s disease trials recorded had a 99.6% failure rate.
In clinical trials, Aduhelm reduced amyloid beta plaques by 59-71% at 18 months of treatment, according to Biogen.
Aduhelm focuses on tackling the accumulation of amyloid beta plaques in the brain: with such plaques a defining pathology of Alzheimer's disease.
Research at Swiss biotech Neurimmune, in collaboration with the University of Zurich, led to the identification of protective anti-amyloid antibodies in healthy elderly people and patients with slowly progressing dementia. Biogen licensed aducanumab from Neurimmune in 2007 under a collaborative development and license agreement. Since October 2017, Biogen and Eisai have collaborated on the development and commercialization of aducanumab globally.
This means the journey for Aduhelm – as with other Alzheimer’s drugs – has been a long one, marking nearly 15 years since Biogen licensed aducanumab from Neurimmune alone (with sizable setbacks along the way).
Aduhelm approval: A 'crucial inflection point in our collective battle'
The FDA’s decision was highly awaited: not least because of the significance of the drug but also because of clinical data that the agency acknowledges are ‘complicated’. But it ultimately approved the drug: with the accelerated approval pathway meaning Biogen is required to conduct a new randomized controlled clinical trial to verify the drug’s clinical benefit.
Biogen's Vounatos says the green light from the FDA represents ‘a crucial inflection point in our collective battle against Alzheimer’s disease.’
“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,” he said.
“More resources will be drawn into research that can help patients through the disease continuum, explore new pathways, and find potential therapy combinations.
“Our industry has been the source of similar types of transformative innovation before. This is exactly how HIV/AIDS and many forms of cancer were changed from untreatable diseases into conditions with viable treatment options. Multiple sclerosis provides a good example. The first therapy introduced in 1993 via accelerated approval set in motion a cycle of innovation that resulted in now more than 20 treatments approved, including six developed by Biogen.
“We are committed to sharing our future insights about Aduhelm with the scientific community as we collect more data from the real-world use of this treatment. In addition, as part of our accelerated approval, Biogen will conduct a confirmatory trial to verify the clinical benefit of Aduhelm.”
While Aduhelm’s approval is a landmark in itself, Vounatos says the drug is just the starting point in treating Alzheimer’s.
“While we launch Aduhelm, we are also investing in more than 30 clinical programs currently under development. These include our investigations into several possible additional treatments for Alzheimer’s disease, as well as other debilitating neurological conditions such as Parkinson’s disease, ALS and stroke. We have spent more than $28bn in research and development since 2003.
“The approval of Aduhelm lays the foundation for creating a new treatment paradigm around Alzheimer’s disease, but what’s needed is far bigger than what any one medicine or any single company can achieve on its own. Today’s milestone is a major step forward in changing Alzheimer’s disease from a disease often perceived as an inevitable consequence of aging to a condition with a new treatment option.”
If we can treat the disease... we need to first detect it
Aduhelm is the first therapy to target and affect the underlying disease process of Alzheimer’s. This means that there is now hope to tackle the disease and stop it progressing, as opposed to simply trying to address symptoms.
For Aduhelm to reach its full potential, therefore, identifying cases early on in the diseases process and widening access to the drug will be crucial, says Vounatos.
“Until now, patients had no treatment option directed at a defining pathology of the disease. Now that a new treatment studied in the early stage of the disease is available, we must collectively shift our focus to early detection, diagnosis and access.”
He wants to see an environment where the detection of Alzheimer’s is ‘as early, widespread and routine as mammograms and colonoscopies.’