Biogen’s Aduhelm (aducanumab) yesterday became the first new treatment approved for the disease since 2003.
- The FDA approved Biogen’s monoclonal antibody Aduhelm for treating Alzheimer’s under the accelerated approval pathway (for drugs that ‘provide meaningful therapeutic advantages over existing treatments’).
- On this path, Biogen must conduct a new clinical trial to verify the drug’s clinical benefit.
- The FDA notes that data for the drug are 'complicated with respect to its clinical benefits', but concluded there is ‘substantial evidence that aduhelm reduces amyloid beta plaques in the brain and the reduction of these plaques is reasonably likely to predict important benefits to patients’.
The Alzheimer's Association – founded in 1980 – is a voluntary health organization in Alzheimer’s care, support and research. Maria C. Carrillo, Ph.D., Alzheimer’s Association chief science officer, told BioPharma-Reporter that the approval of aducanumab is of huge interest to patients and their families.
“This is the first FDA-approved treatment that slows decline due to Alzheimer’s disease," she noted.
"This means individuals may have more time to actively participate in daily life, have sustained independence and hold on to memories longer.
“Aducanumab addresses Alzheimer’s in a new way compared to currently approved drugs. This therapy slows progression of the disease, rather than only addressing symptoms.
"It is the first approved therapy of this type; it demonstrates that removing amyloid from the brain can delay cognitive and functional decline in people living with Alzheimer’s.”
‘Millions of people may be eligible for this therapy’
At the moment, it is difficult to tell how long it will take for a sizeable number of patients to access the treatment. “But we anticipate that demand will be high,” said Carrillo.
“Millions of people may be eligible for this therapy, and our direct experience with our constituents tells us that they are enthusiastic about the chance to have access to new therapies.
“Eliminating barriers to access is our highest priority. The Alzheimer’s Association will do everything in its power to ensure access to the drug, any tests needed during the treatment process, and other associated costs for all who will benefit.
"We know that infusion therapies have been made available under Medicare and insurance for conditions like cancer and multiple sclerosis, so it is reasonable to expect this for aducanumab.”
The Alzheimer’s Association is clear to point out that aducanumab is not a cure: but a drug that slows the progression of the disease.
“In clinical trials, it slowed the rate of cognitive and functional decline," said Carrillo. "This could mean more time for individuals to actively participate in daily life, have sustained independence and hold on to memories longer.
“We know this drug will be of great interest to many, but it is not the only important element of Alzheimer’s treatment and care. The Alzheimer’s Association has made it a priority to partner with health care systems, physicians, and CMS to ensure early and accurate diagnosis, and access to treatment, care management and care planning."
Propelling research efforts forward
As a global leader in Alzheimer’s research, the Alzheimer’s Association works with the scientific community to propel the field of research forward, bringing together government, industry and academia to increase funding and accelerate discovery.
Part of the excitement surrounding the FDA’s approval of aducanumab is the momentum it will push into the area.
“History has shown us that approvals of the first drug in a new category invigorates the field, increases investments in new treatments and encourages greater innovation,” said Carrillo.
The FDA’s accelerated approval of aducanumab, however, will require post-approval clinical trials, in light of the ‘complicated’ data in the Phase 3 trials assessed by the agency. So will this new wave of Alzheimer’s research start straight away, or will innovators and investors remain wary until further data is published?
“Each individual and company will make their own decision based on their risk/reward calculation,” responded Carillo. “That said, we look forward to a significant reinvigoration of the research field – academic and industry - because of this approval.”
In fact, the Alzheimer’s research field had already been growing before yesterday’s decision: so Carrillo hopes the announcement will simply accelerate momentum.
“Driven by Alzheimer’s Association advocacy, federal investment in Alzheimer’s and dementia research funding is $3.1bn annually – six times what it was just a few years ago," she said. "The federal commitment, combined with unprecedented philanthropic support, provides the foundation for an optimistic view of the future.”
What next for Alzheimer's research?
So what does the future of Alzheimer’s research now look like following aducanumab’s approval?
“Eventually, there will be even better treatments of this type, plus new treatments that address the disease in other ways,” says Carrillo.
She describes it as an ‘exciting time’ for Alzheimer’s research: there are currently 126 therapies in clinical trials to treat Alzheimer’s disease (28 are in Phase 3 trials; 74 are in Phase 2 trials; 24 are in Phase 1 trials).
Signficantly, 82.5% of the therapies in clinical trials (104 therapies) are experimental drugs that are designed to change, slow or delay the course of Alzheimer’s disease, rather than just improving symptoms.
Drugs range from those targeting amyloid and tau, as well as other mechanisms including inflammation, microbial approaches, the immune system, the role of the blood-brain barrier, and how brain cells communicate.
“The pipeline now is more diverse than ever before," said Carrillo. "We envision - and are funding clinical trials to make real - multiple therapies that address different aspects of the disease over the entire course of the disease, and that can be combined into powerful and effective combination therapies.
“When combined with better diagnostics that more accurately detect the disease earlier, this will mean more years of full functioning and independent living, and eventually significant risk reduction and effective prevention.”