The Food and Drug Administration approved Biogen Alzheimer’s drug aducanumab, which will be called Aduhelm.
This is the first drug cleared by U.S. regulators to slow cognitive decline in people living with Alzheimer’s and the first new medicine for the disease in nearly two decades.
Aduhelm was approved using the accelerated approval pathway, which can be used for a drug for a serious or life-threatening illness that provides a meaningful therapeutic advantage over existing treatment.
Researchers evaluated Aduhelm's efficacy in three separate studies representing a total of 3,482 patients. The studies consisted of double-blind, randomized, placebo-controlled dose-ranging studies in patients with Alzheimer's disease. Patients receiving the treatment had a significant dose- and time-dependent reduction of amyloid-beta plaque while patients in the control arm of the studies had no reduction of amyloid-beta plaque.
"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, director of the Food and Drug Administration’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.”
Under the accelerated approval, the FDA is requiring Biogen to conduct a new randomized, controlled clinical trial to verify the drug’s clinical benefit. If the trial fails to verify clinical benefit, the FDA may initiate proceedings to withdraw approval of the drug.