Biogen and partner Eisai Co. are ready to get the ball rolling with the FDA-required phase 4 post-marketing study of Aduhelm, the controversial intravenous Alzheimer’s disease treatment approved last June by U.S. regulators.
The companies said they anticipate submitting the final protocol for review to the U.S. FDA in March 2022, and will begin screening patients in May. The study will be a global, placebo-controlled trial, aiming to enroll more than 1,300 early Alzheimer’s disease patients.
The confirmatory trial is a post-marketing requirement of the FDA’s accelerated approval designation.
Part of the debate surrounding Aduhelm was focused on the mismatched results from the drug’s two phase 3 trials — one showed a benefit, while the other did not. Although Biogen presented data suggesting that a discrepancy in dose exposure between patients in the two studies explained the diverging results, not everyone was convinced. But rather than denying the drug, patient advocacy groups argued that the FDA should grant it a conditional approval. On June 7, the agency did just that, saying that Biogen would have to “conduct a new randomized, controlled clinical trial to verify the drug’s clinical benefit,” or it could be pulled from the market.
Biogen and Eisai said their goal is to complete the trial in four years — in half the time the FDA provided.