Yesterday, Biogen’s Alzheimer’s drug aducanamab was approved by the Food and Drug Administration. Also known as Aduhelm, the drug is the first Alzheimer’s treatment to be approved since 2003. Aducanamab could hit the market in as little as ten days, according to Biogen CEO Michel Vounatsos, with hundreds of infusion sites across the United States prepared to administer the treatment.
In a press release acknowledging the FDA’s decision, Alzheimer’s Association Chief Science Officer Maria C. Carillo welcomed the “new era in Alzheimer’s treatment and research” represented by the drug’s approval. Unlike current treatments that only address Alzheimer’s symptoms, namely cognitive decline, aducanamab slows disease progression by removing the characteristic plaques of amyloid protein that accumulate over time in Alzheimer’s patient’s brains.
Aducanamab’s road to FDA approval
Aducanamab was nearly abandoned by Biogen after disappointing results in Phase 3 clinical trials; the company decided to proceed with the drug after a post-hoc analysis of the data suggested positive results in one of the two trials. An advisory committee to the FDA cited this mixed data last November in its official recommendation against approving the drug. In February, the FDA moved to delay its decision on the drug from March to the ultimate June 7th date.
Because of the ambiguity surrounding the treatment’s efficacy, aducanamab has been approved under the FDA’s “accelerated approval” program, meaning that further study is still required and the drug can be taken off the market based on the results of a required confirmatory trial.
Questions and criticisms surrounding the treatment remain
The FDA’s decision does not absolve Biogen from controversy related to the drug. The decision was seen by some in the scientific community as a referendum on the “amyloid hypothesis“, which holds that the buildup of amyloid in the brain is the cause, rather than the byproduct, of Alzheimer’s pathology. Scientists who do not support this hypothesis view the pursuit of amyloid-targeting drugs as a red herring.
Amyloid apostates or not, other detractors worry that aducanamab’s approval will depress public and scientific interest in developing improved drugs for Alzheimer’s, including patient willingness to participate in clinical trials for other treatments. In addition, potential prescribing doctors express skepticism regarding the broad-label approval; aducanamab was approved for all Alzheimer’s patients rather than patients with the early-onset form of the disease studied in the clinical trials.
Critics have also taken aim at aducanamab’s hefty price tag, especially considering the limited data demonstrating the drug’s efficacy. Estimates for what the treatment should cost annually ranged from a $2500-$8300 range put forth by the Institute for Clinical and Economic Review and other outside analysts pricing the drug between $10,000 and $30,000 dollars; yesterday, Biogen announced a $56,000 annual list price for its product. Analysts speculate the high price of this drug, combined with the broad patient population for which the drug was approved, could provoke federal drug pricing reform.
A cause for optimism?
Still, many in the Alzheimer’s community see yesterday’s news as a sign of relief. In Monday’s press release, Alzheimer’s Association president and CEO Harry Johns expressed hope that aducanamab may allow patients to “have more time to actively participate in daily life, have sustained independence and hold on to memories longer.”
“We can experience longer — the relationships we hold most dear — our families and friends,” he added.