TThe US Food and Drug Administration has approved aducanumab, a monoclonal antibody under the brand name Aduhelm, for the treatment of Alzheimer’s disease in early-stage patients. With today’s announcement (June 7th), the FDA has approved a drug for the treatment of neurodegenerative disease for the first time in 17 years.
“After so many disappointments with possible treatments, the Alzheimer’s community now knows that this mortal enemy is finally on the line. Science and time are finally on our side, ”says a statement from UsAgainstAlzheimer’s, an interest group.
That enthusiasm is dampened by a number of experts who say the drug does not make a significant difference to patients. Caleb Alexander, an FDA advisor and researcher at Johns Hopkins University, recommended the drug’s approval. “The FDA gets the respect it has for having regulatory standards that are based on solid evidence. In this case, I think they passed the product, ”he told the Associated Press.
Aducanumab clears amyloid plaques in the brain, and data from an early clinical study showed it to be remarkably effective at doing this. “Overall, this is the best news we’ve received in my 25 years of clinical research,” said Stephen Selloway, a physician at Butler Hospital in Providence, Rhode Island who led the study when these results were released in 2016.
However, later results from two Phase 3 studies showed that aducanumab did not affect recipients’ mental abilities sufficiently, and in 2019 Biogen and Eisai, the drug’s developers, stopped the studies. Half a year later, and after looking back at the data, the companies found that participants who received the highest dose of the drug had less cognitive decline, along with fewer amyloid and tau proteins in their brains than participants who did had received a placebo.
The companies decided to seek regulatory approval, but in November 2020 the FDA panel that Alexander sits on recommended against it, noting that only one of the two clinical trials showed any benefit.
The agency went through with the approval, but with conditions. How STAT notes that the approval is based on aducanumab’s ability to eradicate amyloid plagues, and companies need to continue testing and demonstrate clinical benefits in order to keep the drug in the market. “With the conditional approval of the drug, the agency deviated from decades of regulatory precedents.” STAT reports “Setting a new benchmark for treatments with considerable potential but unproven benefits – a standard that could be applied to other devastating diseases as well.”