Adcomm member who warned about risk of approving Biogen's aducanumab has resigned following historic OK — reports
One of the 11 experts the FDA had convened to discuss Biogen’s aducanumab — before overriding their recommendation and approving the Alzheimer’s drug — has resigned from the panel, STAT News and Pink Sheet reported.
Joel Perlmutter, a neurologist at Washington University in St. Louis who’s been on the Peripheral & Central Nervous System Drugs Advisory Committee roster since 2015, told STAT that he quit on Monday “due to this ruling by the FDA without further discussion with our advisory committee.”
The adcomm’s opinion was near-unanimous: When asked whether it is reasonable to consider the positive Phase III Biogen submitted “as primary evidence of effectiveness” for the treatment of Alzheimer’s disease, 10 voted no, none said yes and one abstained. During the full-day session last November, experts excoriated both the company and the agency for what they saw as insufficient data and a biased presentation of information.
At one point, Perlmutter warned that an approval for aducanumab could push back a truly effective treatment “for more than a couple of years, for many years.”
“Alzheimer’s treatment is a huge, urgent, unmet need, but I also think if we approve something where the data is not strong, we have a risk of delaying good treatment,” he was quoted as saying.
The FDA’s historic approval of aducanumab was a resounding victory for Biogen, which stands to gain billions in revenue from the drug, now dubbed Aduhelm, with a broad label priced at $56,000 per year. While patient advocacy groups and some leading neurologists hailed the milestone, others decried possible false hope and expressed concerns about the precedent that regulators have now set regarding everything from the validity of beta-amyloid as an Alzheimer’s target to the threshold for clinical evidence.
Biogen has nine years to complete the confirmatory trial.
But the FDA is now back-tracking on that timeline, saying in an emailed statement to Endpoints News, “We consider the nine-year timeline a conservative estimate, which is an appropriate starting point for development of the draft protocol from the sponsor for its global confirmatory study. The agency believes it’s important that sponsors set achievable clinical trial target timelines. We fully appreciate the high level of interest in getting these answers quickly and will carefully monitor trial progress and support efforts to complete this trial in the shortest possible timeline.”
FDA also says it will continue to work actively with Biogen in the months ahead on plans for this confirmatory trial, including review and discussion of the assumptions for the proposed timeline.
“We expect the sponsor to commit all resources needed to move this trial forward as effectively as possible, with the aim of completing the trial as soon as is feasible, while assuring the quality of the data and the robustness of the results. Alzheimer’s trials take time to complete,” FDA said.