FDA Sharply Narrows Recommended Use of Biogen's New Alzheimer’s Drug

The Food and Drug Administration on Thursday dramatically narrowed its recommended usage guidelines for Alzheimer’s drug Aduhelm, suggesting that only patients with milder forms of the disease should take it. The change follows sharp criticism of the agency’s decision last month to approve the treatment for all Alzheimer’s patients — and it could greatly reduce both the number of patients eligible to receive Aduhelm and the potential costs of the drug to Medicare, which is expected to pay for most prescriptions.

The original prescribing information, which said that the drug could be appropriate for anyone with Alzheimer’s, meant that about six million Americans could get it. The revised label recommends that the drug be given to patients with mild cognitive impairment or mild dementia, a smaller group that matches the population in drugmaker Biogen’s clinical trials. Under the new label, some 2 million Americans reportedly may be eligible.

The new label also says, “There are no safety or effectiveness data on initiating treatment at earlier or later stages of the disease than were studied.”

The original “accelerated” approval of Aduhelm had set of waves of criticism given that the benefits of the drug remain uncertain and that its list price — $56,000 a year — meant that covering the drug could add billions of dollars in costs to Medicare. Doctors may still prescribe the drug off-label for patients with more advanced Alzheimer’s, but the new label could allow Medicare to restrict access to early-stage patients, lowering the potential cost.

“We don’t expect the new Aduhelm label to impact peak sales for this product,” SVB Leerink analyst Marc Goodman said in a note to clients, according to The Wall Street Journal. But it “should help narrow the patient population that seeks treatment and make it somewhat easier for physicians to ‘say no’ to many patients that shouldn’t be candidates for usage in the first place” and “help the payers for the same reason,” he added.

Changing a drug’s prescribing label drug so soon after its approval is highly unusual. Biogen and the FDA described the change as a clarification, but some doctors said that Thursday’s change only raises more questions about the FDA’s process, and many suggest that the revisions should have been more extensive.

The New York Times reports: “Many experts say that the drug’s label should not only narrow the use of Aduhelm to mild stages of the disease, but should also require two other strict conditions of the clinical trials: that eligible patients have evidence of high levels of a key protein, amyloid, in their brains, and that people with certain medical conditions (called ‘contraindications’) should be prevented from taking the drug, or at least designated as a high-risk group, because it can cause brain swelling and brain bleeding.”