Q&A: Aducanumab, one of 121 experimental AD drugs in clinical trials, awaits FDA approval
In early November, the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee met to discuss a Biologics License Application for aducanumab, an anti-amyloid antibody in development for Alzheimer’s disease.
The advisory committee ultimately decided not to recommend an approval for the agent, according to Joanne Pike, DrPH, chief strategy officer for the Alzheimer’s Association. As a result, the FDA review process for aducanumab is ongoing. A decision regarding the approval of the Biologics License Application for the agent is now expected by June 7, 2021, according to a new release issued by Biogen, the maker of aducanumab, following the advisory committee meeting.
Healio Neurology spoke with Pike to learn more about aducanumab, which is administered intravenously once a month and works by clearing some of the plaque materials that may cause cell death and tissue loss caused by AD, and other agents in the pipeline for this disease.
Q: How does aducanumab work to slow the progression of AD?
A: Aducanumab is not a cure for AD. Based on the publicly available clinical trial data, it appears to slow the rate of cognitive and functional decline and therefore may help preserve patients’ independence longer. Aducanumab would be the first drug that changes the underlying course of the disease, rather than only addressing the symptoms like the currently approved AD drugs. It is also the first therapy to demonstrate that removing amyloid from the brain may delay clinical decline with benefits to both cognition and function in people living with AD.
Q: Where does aducanumab currently stand with the FDA?
A: The FDA has not yet made a decision on aducanumab. The FDA review process included an advisory committee review. That committee, after reviewing the data submitted by Biogen, recommended against an approval. However, the committee's votes are nonbinding; in fact, in recent years, the FDA has approved many drugs based on a single phase 3 trial. There are multiple examples of this for cancer therapies and spinal muscular atrophy.
While the trial data has led to some uncertainty in the scientific community, this must be weighed against the certainty of what this disease will do to millions of Americans without treatment. For example, requiring completion of an additional phase 3 trial would deny broad access for up to 4 years while it is completed. A 4-year delay is too long to wait for millions of Americans facing a progressive, fatal disease. A 4-year delay is too long to wait for millions of American caregivers.
Q: What are the next steps for the development of aducanumab?
A: Aducanumab is still under review by the FDA. We appreciate the agency’s rigorous scientific review and prioritization of the health and safety of our constituents.
The Alzheimer’s Association will continue to advocate for the FDA approval of aducanumab on behalf of the millions of people living with, and caring for, people with AD. There is a significant need to offer relief and support to the millions of Americans impacted each day by the crushing realities of AD, a progressive and fatal disease. Given the devastating toll of this disease, the publicly released data justify approval accompanied by a phase 4 postmarketing surveillance study. The Alzheimer’s Association urges the FDA’s approval of this agent.
Q: What other agents are in development for AD?
A: There are currently 121 experimental drugs in clinical trials to treat AD: 29 are in phase 3 trials, 65 are in phase 2 trials and 27 are in phase 1 trials. Of the 121 drugs, there are 97 experimental drugs that are designed to change, slow or delay the course of AD, beyond just improving symptoms. Compared to a survey of the 2019 drug pipeline, there are now more experimental drugs targeting treatment pathways beyond amyloid or tau.
Driven by Alzheimer’s Association advocacy, federal investment in AD and dementia research funding is $2.8 billion annually, a more than 6-fold increase from just 10 years ago. The federal commitment, combined with unprecedented philanthropic support, provides the foundation for an optimistic view of the future — which is needed, because there is much work to be done.
Whatever the outcome with aducanumab, we need to address treatment of AD and all other dementias from multiple angles. AD is a complex disease that will very likely require a complex treatment. Examples of the Alzheimer's Association's leadership in research and diversifying the treatment pipeline include:
The Accelerating Medicines Partnership-Alzheimer’s Disease (AMP-AD), a large-scale effort involving government, nonprofits, including the Alzheimer’s Association, and the drug development industry. It is focused on verifying new treatment targets.
The Alzheimer’s Association’s Part the Cloud initiative is funding innovative drug trials focusing on new treatment targets. To date, $54 million has been awarded to 55 clinical trials.
- Biogen. Update on FDA Advisory Committee’s meeting on aducanumab in Alzheimer’s disease. https://investors.biogen.com/news-releases/news-release-details/update-fda-advisory-committees-meeting-aducanumab-alzheimers. Accessed Dec. 1, 2020.
- Healio Interviews.