Newly approved Alzheimer’s drug raises serious concerns
United States health officials have approved the first new drug to treat Alzheimer’s in twenty years.
But independent advisors and some Alzheimer’s experts have said the approval comes without enough evidence that the drug can help patients with the brain-destroying disease.
The Food and Drug Administration said it approved the drug, aducanumab, based on results that its benefits for Alzheimer’s patients were “reasonably likely”.
Aducanuman, which will go by the brand name Aduhelmm, is produced by American biotechnology company Biogen and is to be administered as a monthly infusion.
As the first approved treatment to directly attack the disease process of Alzheimer’s instead of just treating the symptoms like anxiety and insomnia, the drug is intended to slow the cognitive decline in those with mild memory and thinking problems.
The drug is thought to reduce harmful clumps of plaque in the brain in order to slow the progression of the deadly disease.
Why this matters
With the approval of the treatment despite limited evidence that it works, experts are concerned it could set a precedent for the approval of future experimental therapies that only show incremental benefits.
Currently only five medications have been approved by the FDA for patients with Alzheimer’s, though these only delay cognitive decline for several months.
As a result, patient advocacy groups lobbied for approval to expand the number of treatments available of Aduhelm, with other more-promising candidates likely to be three to four years away from potential approval.
But, the FDA advisory committee, as well as an independent think tank and several prominent experts - including Alzheimer’s doctors who were involved in clinical trials of the drug - have said the evidence raises doubts about its effectiveness. Concerns have also been raised about the small benefits being outweighed by the risk of swelling or bleeding in the brain that occurred during the trials.
The risk of bleeding is also heightened for those with the APOE4 gene, which is associated with late-onset Alzheimer’s.
Caleb Alexander, an FDA advisor who recommended against the drug’s approval, said he was “surprised and disappointed”.
“The FDA gets the respect that it does because it has regulatory standards that are based on firm evidence. In this case, I think they gave the product a pass,” said Dr Alexander, a medical researcher at John Hopkins University.
Dr Patrizia Cavazzoni, the FDA’s director of the Centre for Drug Evaluation and Research wrote on the agency’s website: “The data included in the applicant’s submission were highly complex and left residual uncertainties regarding clinical benefit,”
But, she said the agency had approved the drug through a program called accelerated approval, which is designed to provide earlier access to therapies for diseases with few treatments, “and where there is an expectation of clinical benefit despite some residual uncertainty regarding the benefit.”
Dr Cavazzoni also told reporters: “The data supports patients and caregivers having the choice to use this drug.”
What happens next
Though approved, the FDA has required the drug manufacturer to conduct a follow-up study to confirm the benefits. If this study fails to show the drug's effectiveness, the FDA can - but isn’t required to - withdraw the drug from the market.
Biogen has said it aims to complete the follow-up trial by 2030.
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