On Monday, the U.S. Food and Drug Administration (FDA) approved Aduhelm (aducanumab) for the treatment of Alzheimer’s disease.
Aduhelm was approved using the accelerated approval pathway, the FDA noted, which can be used for a drug for a serious or life-threatening illness that provides a meaningful therapeutic advantage over existing treatments.
Accelerated approval can be based on the drug’s effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients, with a required post-approval trial to verify that the drug provides the expected clinical benefit.
“Alzheimer’s disease is a devastating illness that can have a profound impact on the lives of people diagnosed with the disease as well as their loved ones,” said Patrizia Cavazzoni, M.D., director of the FDA‘s Center for Drug Evaluation and Research. “Currently available therapies only treat symptoms of the disease; this treatment option is the first therapy to target and affect the underlying disease process of Alzheimer’s. As we have learned from the fight against cancer, the accelerated approval pathway can bring therapies to patients faster while spurring more research and innovation.”
The drug, Aduhelm, a monthly infusion priced at $56,000 per year, was approved this week despite weak evidence that it helps patients.
Since the approval Monday, three FDA advisors have resigned over the approval of Aduhelm in protest at the agency’s decision to approve Biogen Inc’s treatment of Alzheimer’s disease despite the committee’s recommendation against doing so. These include Washington University neurologist Dr. Joel Perlmutter, who resigned from the FDA’s advisory committee on Tuesday.
He was followed Wednesday by Mayo Clinic neurologist Dr. David Knopman and Professor of Medicine at Harvard Medical School, Aaron Kesselheim on Thursday.
Dr. Knopman said, “I was very disappointed at how the advisory committee input was treated by the FDA. I don’t wish to be put in a position like this again.”
Kesselheim, who had served on the FDA’s advisory committee for nervous system drugs since 2015, told Reuters: “My rationale was that the FDA needs to re-evaluate how it solicits and uses the advisory committees … because I didn’t think that the firm recommendations from the committee in this case … were appropriately integrated into the decision-making process”.