The US Food and Drug Administration on Monday approved the use of drug aducanumab, an experimental drug, to treat the early phases of Alzheimer’s disease – despite the FDA advisory committee issuing in 2020 that enough evidence is not available to support the effectiveness of the drug.
The drug was developed for patients with subtle cognitive impairment and not severe dementia. Apart from easing symptoms, the drug intends to slow down the progression of Alzheimer’s disease.
The FDA has not approved a novel therapy for Alzheimer’s disease since 2003.
Using its “accelerated approval” program, the FDA approved aducanumab or Aduhelm, which allows for earlier approval of a drug for a serious and life-threatening disease even though more study may be required to test the drug’s benefits.
On Monday, Dr. Patrizia Cavazzoni announced that there have been numerous public debates on whether or not Aduhelm should be approved and as it is a case of interpreting scientific data, the expert community has offered different perspectives.
The FDA decided to approve Aduhelm for patients with Alzheimer’s disease after coming to the conclusion that the benefits of the drug exceed the risks of therapy. The data has been interpreted differently by different experts regarding the drug but FDA followed its usual course of action while making decisions related to such cases.
Cavazzoni said that even under accelerated approval, the drug will be studied by way of conducting “post-approval studies” by drug companies. These studies are known as phase 4 confirmatory trials that verify if treatments have clinical benefits. In case the confirmatory trial fails to confirm the benefit of the drug, it could be removed from the market by the FDA.
Regarding evidence of the drug’s effectiveness, FDA’s Peripheral and Central Nervous System Drugs Advisory was asked to vote on the matter. When asked if it was reasonable to consider data from a single positive study as the main evidence of Aduhelm’s effectiveness for the treatment of early stages of Alzheimer’s disease, none of the committee members voted yes, ten members voted no and some were uncertain.
The committee’s response was then left with the FDA to decide whether to approve the drug or slow down for now.
According to Cavazzoni, most studies indicated Aduhelm’s consistency and efficiency in reducing the level of amyloid plaques in the brain in a dose. A reduction in clinical decline due to reduction in amyloid plaque is expected.
Dr. Richard Isaacson, director of the Alzheimer’s Prevention Clinic at Weill Cornell Medicine and NewYork-Presbyterian, who tested patients in the primary aducanumab clinical studies revealed that the drug will be used for the treatment of earliest symptomatic phase of the disease called mild cognitive impairment due to Alzheimer’s, the pre-dementia period but not severe dementia.
According to experts, expectations should be tempered and it must be explained to people that the drug is meant for only the earliest symptomatic phases. It won’t benefit in case of a severe Alzheimer’s patient.