FDA approves first new Alzheimer’s disease drug in nearly 20 years
(CNN) – In a controversial decision, the US Food and Drug Administration approved the use of an experimental drug used for early phases of Alzheimer’s disease Monday.
The drug aducanumab was approved despite an FDA advisory committee concluding last year that there is not enough evidence to support the effectiveness of the treatment, CNN reports.
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.
The FDA has not approved a novel therapy for Alzheimer’s disease since 2003.
The FDA approved aducanumab, also known as Aduhelm, using its “accelerated approval” program, which allows for the earlier approval of a drug for a serious or life-threatening illness even though more study into the drug’s benefits may be needed.
In November, the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee was asked to vote on several questions about evidence of the drug’s effectiveness. In response to a question about whether it was reasonable to consider data from one positive study as the primary evidence of aducanumab’s effectiveness for the treatment of early Alzheimer’s disease, none of the committee members voted yes — 10 voted no and one was uncertain.
The committee’s opinions were then left with the FDA as the agency mulled whether to approve the drug or pump the brakes.
The pharmaceutical company Biogen and its Japanese partner Eisai developed aducanumab, administered through intravenous infusion to treat early Alzheimer’s disease. The drug was developed for patients with mild cognitive impairment, not severe dementia.
Dr. Richard Isaacson, director of the Alzheimer’s Prevention Clinic at Weill Cornell Medicine and NewYork-Presbyterian in New York, who had patients in the original aducanumab clinical studies, told CNN the drug targets the earliest symptomatic phase of the disease, called mild cognitive impairment due to Alzheimer’s. Treatment of the pre-dementia period was the focus of the FDA’s decision.
“We have to really temper expectations and explain to people that this drug is meant for the earliest symptomatic phases,” he said. “It pains me to say this but if I have a severe Alzheimer’s patient that can no longer speak or interact much with others and their family member is begging me to give them this drug, I won’t be able to do it.”
Aducanumab’s road to FDA approval has been rocky — and controversial.
In March 2019, Phase 3 clinical trials of aducanumab were discontinued because a futility analysis found the trials were unlikely to meet their primary goals at completion.
“The futility analyses showed the studies were most likely to fail,” Isaacson said.
Several months later, Biogen announced that a new analysis, which included more patients, showed that those who received high doses aducanumab experienced a reduction in clinical decline in one trial.
The company noted in FDA briefing documents last year that patients treated with high-dose aducanumab showed 22% less clinical decline in their cognitive health at about 18 months — meaning the progression of their early Alzheimer’s disease slowed — compared with those who received a placebo.
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