aduhelm label, aducanumab

FDA Limits the Use of Aduhelm After Criticisms of Broad Approval

By Nicholas Chan | July 8th, 2021

Following sharp criticisms of the FDA’s broad approval of Aduhelm for all patients with Alzheimer’s, the agency has narrowed the drug’s label to those with early Alzheimer’s symptoms.

UPDATE: 3 March 2024, 8:28 P.M. ET. In February 2024, Biogen took Aduhelm off the market, citing financial concerns. Although the drug did receive accelerated, conditional FDA approval for the treatment of early Alzheimer’s disease in 2021, it is no longer available to new patients. The company announced it would sunset trials in May 2024 and cease supplying the drug to current patients in November 2024.

The FDA has revised the label of the new Alzheimer’s drug Aduhelm (aducanumab), recommending its use for people who are in the early stages of the disease. The clarification comes after confusion over the drug’s label, which previously indicated it could be prescribed to all people diagnosed with Alzheimer’s disease.

In a highly unusual move, the agency reissued the label to specify that Aduhelm, a drug designed to clear the hallmarks of Alzheimer’s known as amyloid plaques, should be prescribed to patients with mild cognitive impairment (MCI) or mild Alzheimer’s dementia. 

“Since the agency approved Aduhelm, prescribers and other stakeholders have expressed confusion regarding the intended population for treatment,” an FDA representative wrote to Being Patient in an email. “A key question has been whether the Aduhelm prescribing information supports the treatment of patients at all stages of Alzheimer’s disease. Hearing these concerns, the FDA determined that clarifications could be made to the prescribing information to address this confusion.” 

Daniel Gibbs, previously a participant in Aduhelm’s clinical trial and a retired neurologist, said the drug has not been tested in patients with advanced Alzheimer’s, and the revised label will help curtail Aduhelm’s prescriptions from those in the later stages of the disease. 

“The changes in the prescribing information to limit the use of Aduhelm to those with MCI or mild dementia due to Alzheimer’s disease are welcome and very important,” Gibbs wrote to Being Patient in an email. “This should limit inappropriate use in more advanced disease for which it is unlikely to be beneficial and may be more dangerous.” 

He added, “I strongly feel that Aduhelm should not be used in moderate and severe Alzheimer’s outside of a clinical trial until safety and efficacy have been studied in these groups.”

Since the FDA’s approval of Aduhelm last month, experts have raised concerns about the disconnect between the drug’s initial label and the criteria of its clinical trials. 

“There’s really no evidence that it (Aduhelm) works for later stage disease and it probably doesn’t work for later stage disease because based on the biology of what we know about Alzheimer’s, the amyloid process pretty much peaks during the earlier stage,” Dr. Stephen Salloway, a principal investigator of the aducanumab trial and the director of Butler Hospital’s Memory and Aging Program, told Being Patient on the day of Aduhelm’s approval.  

Want to learn more about clinical trials
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Check out the Lilly Trial Guide.

Given that Alzheimer’s is a progressive disease, the FDA stated Thursday that ongoing treatment of Aduhelm may still help some patients as they progress from MCI or mild dementia to the later stages of Alzheimer’s. “Prescribers should evaluate their individual patient’s response to treatment with aducanumab and determine if there is potential for benefit with continued treatment,” the agency’s representative said.

Meanwhile, some experts have noted that Medicare spending would balloon under Aduhelm’s previous label as patients with more progressed Alzheimer’s may be motivated to pursue treatment, even though there isn’t evidence of its safety and efficacy for those in the later stages of the disease. The updated label could guide Medicare and private insurers toward restricting the coverage of Aduhelm, which costs $56,000 annually per patient.

However, Gibbs said another shortcoming remains in the updated label. It does not limit Aduhelm treatment only to those with biomarkers of Alzheimer’s pathology, such as positive amyloid PET scans or amyloid levels from lumbar punctures. Blood tests for amyloid will also most likely be available within a few years, he added, which present a more accessible and cheaper method of identifying Alzheimer’s hallmarks. But “without requiring evidence of Alzheimer’s pathology, the drug will be used in many people at great cost with non-Alzheimer’s dementia for which it will not offer any benefit but with possible as yet unknown side effects,” Gibbs said. 

As of press time, the FDA did not respond to concerns about the lack of biomarker testing specifications in Aduhelm’s label. 

Aside from controversies over the drug’s label, the FDA’s approval has been marked by criticisms over issues that range from Aduhelm’s efficacy to cost. Clinicians urge patients and families to hold thorough discussions with their health care providers before pursuing treatment. 

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