FDA approve aducanumab (Aduhelm) for treatment of Alzheimer’s disease
This amyloid beta-directed antibody has been evaluated in 3 double-blind, randomised, placebo-controlled dose-ranging studies (n=3482), which reported significant dose-and time-dependent reduction of amyloid beta plaque vs. no reduction with control arm
Source:
US Food and Drug Administration
SPS commentary:
Under the accelerated approval provisions, which provides earlier access to the treatment in US, the FDA is requiring the company, Biogen, to conduct a new RCT to verify the drug’s clinical benefit.
Aducanumab is described as a first-of-its-kind treatment approved for Alzheimer’s disease; the first new treatment approved in US since 2003 and the first therapy that targets the fundamental pathophysiology of the disease.
News reports note scientists are divided over potential impact of this treatment because of uncertainty over the trial results. In March 2019, late-stage international trials of aducanumab were halted when analysis showed the drug, given as a monthly infusion, was not better at slowing the deterioration of memory and thinking problems than placebo. Later that year, Biogen analysed more data and concluded the drug did work, as long as it was given in higher doses.
In Oct 2020, the European Medicines Agency confirmed it had accepted for review, the Marketing Authorization Application for aducanumab.