Few days ago the FDA approved Aduhelm (adacanumab) as a new treatment for Alzheimer’s disease. This will be a new treatment for Alzheimer’s disease which can also be referred to as senile dementia.
Alzheimer’s disease is a progressive disease that destroys the memory and other important mental functions. Prior to this announcement, Alzheimer’s had no cure and was only managed symptomatically.
However amidst the announcement, some experts still suggest that there’s not enough evidence that Aduhelm can address cognitive symptoms of Alzheimer’s disease.
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Arguments center on the fact that the drug was developed for patients with mild cognitive impairment, not severe dementia, and intended to slow progression of Alzheimer’s disease — not just ease symptoms.
There was also the cost factor. It was announced by Biogen (the pharmaceutical company behind Aduhelm) and it’s Japanese partner Eisai that an infusion of Aduhelm is required once every four weeks to derive maximum benefits. The cost per infusion is about $4,312, making the annual cost around $56,000.
FDA’s Reaction and Approval.
In a statement by Dr. Patrizia Cavazzoni, Director, FDA Center for Drug Evaluation and Research, it was agreed that the FDA indeed approved Aduhelm as a new treatment for Alzheimer’s disease using the Accelerated approval pathway.
In the Accelerated approval pathway, the FDA approves a drug for a serious or life threatening illness that may provide meaningful therapeutic benefit over existing treatments.
This often happens when the new drug is shown to have an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients. There may however be some uncertainty about the drug’s overall clinical benefit.
Aduhelm is the first new treatment for Alzheimer’s disease approved since 2003. Perhaps more significant is the fact that Aduhelm is the first treatment directed at the underlying pathophysiology of Alzheimer’s disease—the presence of amyloid beta plaques in the brain.
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.
Results of Clinical Trials with Aduhelm.
The late-stage development program for this new treatment for Alzheimer’s disease consisted of two phase 3 clinical trials. One study met the primary endpoint, showing reduction in clinical decline. The second trial did not meet the primary endpoint.
Meanwhile, in all studies in which it was evaluated that Aduhelm consistently and very convincingly 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.
As one would also expect a phase 4 trial is also needed. In a stage like this, drug companies are required to conduct post-approval studies to verify the anticipated clinical benefit. If the confirmatory trial does not verify the drug’s anticipated clinical benefit, FDA (and even other regulatory bodies) could remove the drug from the market.
The Ruins of Alzheimer’s Disease.
There is a gradual and cumulative devastation that Alzheimer’s disease causes. Patients lose their memory and cognitive functioning over time. In late stage disease, people can no longer hold a conversation or respond to their environment.
On an average a person with Alzheimer’s disease lives four to eight years after diagnosis, but some patients can live up to 20 years with the disease. Therefore, the need for a new treatment for Alzheimer’s disease can longer be overemphasized.
Data shows that only in America more than 6 million people are living with Alzheimer’s disease and this number is expected to grow as the population ages. Alzheimer’s is the sixth leading cause of death in the United States.
Although the Aduhelm data are complicated with respect to its clinical benefits, FDA has determined that there is substantial evidence that this new treatment for Alzheimer’s disease reduces amyloid beta plaques in the brain and that the reduction in these plaques is reasonably likely to predict important benefits to patients.
The best we can all do therefore is to be hopeful that this new treatment for Alzheimer’s disease proves to be effective. This is because it will go a long way in improving the quality of life of the aged population and also their care givers.
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