PERTH, Australia – Digital cognitive assessment technology company Cogstate Ltd. saw its stock skyrocket 60% on the heels of FDA accelerated approval of Biogen Inc./Eisai Co. Ltd.’s Alzheimer’s disease drug Aduhelm (aducanumab). Cogstate and Eisai signed a deal in October 2020 that granted Eisai global development rights and exclusive commercialization rights of all cognitive function tests developed by Cogstate.

LONDON – Controversy over the quality of the data supporting approval of Biogen Inc.’s Aduhelm (aducanumab), and FDA’s requirement for a phase IV study to demonstrate real-world evidence of the effectiveness of the drug, has put the spotlight on the need for better dementia diagnostics, both to stratify trials and to track any reduction in clinical decline.

Biogen Inc.’s pricing of its newly approved Alzheimer’s drug, Aduhelm (aducanumab), has made it the latest bull’s eye for lawmakers and advocacy groups targeting U.S. drug prices, especially given the controversy surrounding the drug’s approval, which has resulted in the resignation of three of the 11 members of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee.

Of all the controversies surrounding the FDA, the agency’s reliance on user fees and its use of accelerated review of therapies might be the most consistent sources of public angst. Coleen Klasmeier, a partner of Sidley Austin LLP, told BioWorld that while she is not particularly concerned about regulatory capture stemming from FDA reliance on user fees, it may be appropriate to ask whether the drug premarket review process leaves FDA staff with more confidence in a new drug application than the data would seem to suggest.

Developers of blood tests for the early diagnosis of Alzheimer’s disease (AD) are poised to see a spike in demand, following the FDA’s accelerated approval on Tuesday of Cambridge, Mass.-based Biogen Inc.’s AD drug treatment Aduhelm (aducanumab). The FDA’s decision paves the way for AD assays to move beyond aiding in drug development toward addressing ongoing issues with undiagnosed cases.

During a conference call with investors, Biogen Inc. officials defended the price of Aduhelm (aducanumab) for Alzheimer’s disease (AD), with CEO Michel Vounatsos saying the $56,000 per year price tag was based on “the value it is expected to bring to patients, caregivers, and society.” 

By granting accelerated approval for Biogen Inc.’s Aduhelm (aducanumab) for Alzheimer’s disease (AD), the FDA is “essentially confirming that the beta-amyloid hypothesis has been validated,” Mizuho analyst Salim Syed said in an alert to investors. Shares of Cambridge, Mass.-based Biogen (NASDAQ:BIIB) closed $395.85, up $109.71, or 38.3%, as Wall Street hailed the first new AD therapy to reach the market since 2003.

Public biopharmaceutical companies did manage to attract some investors off the sidelines in May with medical conference season getting into full gear. However, it was generally another lackluster month, with the Nasdaq Biotech index dropping 2% in the period in contrast to the broader markets with the Dow Jones Industrial Average growing by 2%. The sector, however, could get a significant boost in the next few days if the FDA gives the green light to Biogen Inc. and Eisai Co. Ltd.’s experimental Alzheimer’s disease therapy, aducanumab, a recombinant chimeric human IgG1 monoclonal antibody targeting beta-amyloid, that could be the first disease-modifying therapy for an indication that has seen no novel therapies approved in more than 15 years.