The FDA approved aducanumab (Aduhelm, Biogen) for the treatment of Alzheimer’s Disease. Use of aducanumab was not limited to patients with early Alzheimer’s, similar to the patient populations in the ENGAGE and EVOLVE trials. Rather it was approved for use in any patient with Alzheimer’s disease.
The prescribing information for aducanumab contains a warning for amyloid-related imaging abnormalities (ARIA). ARIA usually presents as transient, symptomatic swelling in the brain. But some patients may develop headache, confusion, dizziness, vision changes, or nausea with ARIA. In addition to ARIA, the most common adverse effects include headache, fall, diarrhea, and confusion/delirium/altered mental status/disorientation.
A review by the FDA’s Division of Neurology concluded that positive data from Emerge and a Phase II trial provided substantial evidence of effectiveness to support approval of aducanumab. However, a review by the FDA’s Office of Biostatistics concluded that a new trial was needed, because of the conflicting evidence seen in EMERGE and ENGAGE.
The FDA Peripheral and Central Nervous System Drugs Advisory Committee voted 0 yes, 10 no and 1 uncertain on whether there was enough evidence to recommend approval of aducanumab for the treatment of Alzheimer’s disease in November 2020. The FDA extended the review date for aducanumab by three months (March to June) to allow additional time to review new data Biogen had submitted to satisfy an FDA information request.
As part of the approval, the FDA is requiring Biogen, to conduct a new clinical trial to verify the aducanumab’s clinical benefit.
Biogen set the annual price for aducanumab at $56,000 a year, which is much higher than ICER’s cost effectiveness estimates of $2,500 to $8,300. ICER estimated that even in the most optimistic case, where only data from the positive EMERGE trial is used, the limited cognitive improvement would only support a price range of $11,100 to $23,100 per year.
The Prescribe Right Pharmaceutical Pipeline Tracker added drug reviews for drugs with PDUFA dates this spring. In our review of aducanumab, we give an in-depth analysis of the ENGAGE and EMERGE trials, the discontinuation of the trials after a futility analysis, their resurrection after a new analysis, the FDA review of the clinical data and the limitations of current Alzheimer’s therapy. We also include a review of adverse effects, votes by the FDA Peripheral and Central Nervous System Drugs Advisory Committee, an editorial in JAMA by three committee members, a look at the cost of other biologic drugs to treat neurological conditions and a summary of the ICER cost effectiveness analysis. As always, the source references for the review are included along with summaries of each article. As a subscriber, you will have access to these reviews, along with monographs for over 900 drugs to allow you to know what is coming, be ready to discuss controversial new drugs and plan for budget busting new therapies.
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