In the lead-up to European regulators' December decision on Aduhelm's approval for use in the European Union, an expert panel declined to give the controversial Alzheimer's drug its vote of confidence, advising against approval in the E.U.
UPDATE: 3 March 2024, 8:25 P.M. ET. In February 2024, Biogen took Aduhelm off the market, citing financial concerns. Although the drug did receive accelerated, conditional FDA approval for the treatment of early Alzheimer’s disease in 2021, it is no longer available to new patients. The company announced it would sunset trials in May 2024 and cease supplying the drug to current patients in November 2024.
Anti-amyloid drug Aduhelm has been mired in controversy surrounding its U.S. approval and its questionable efficacy. After news that the drug underperformed estimated sales by millions of dollars, this week a panel of European regulatory advisors voted against recommending its approval. While the panel does not have the final say, it is another blow to drugmaker Biogen, which has struggled to sell their new drug in the United States.
This vote will be considered by the European Medicines Agency (EMA), which will make the final decision in approving new drugs in the E.U. this December. Similarly, an expert panel in the U.S. previously recommended against Aduhelm’s American approval, though it was ultimately approved by the Food and Drug Administration.
“Given the existing data and controversy attached to the drug, we see no reason why there would be a divergence [in EMA’s decision],” said Wedbush analyst Laura Chico. Biogen’s top scientist, who spearheaded Aduhelm’s development, Alfred Sandrock — whose off-the-books meetings with FDA officials are currently under federal investigation — also announced his surprise retirement on November 15th, two days before the European advisory panel’s vote. It is unclear whether this retirement is related to the vote.
Biogen did not anticipate an endorsement from this panel, releasing a statement before the vote: “While we are disappointed with the trend vote, we strongly believe in the strength of our data and that aducanumab has the potential to make a positive and meaningful difference for people and families affected by Alzheimer’s disease,” said Priya Singhal, Biogen head of global safety and regulatory sciences and interim head of research and development.
A key issue considered by the EMA is the cost-effectiveness of the drug, weighing any potential side effects against its benefits. In the U.S., the drug is available at a hefty $56,000 USD per year, creating complexity around insurance coverage and spurring a rise in Medicare premiums. The Institute for Clinical and Economic Review calculated that it would need to slash its high price tag to be cost-effective.
If approved, drugmakers would need to negotiate insurance reimbursement for Aduhelm with different countries in the E.U. independently. Other anti-amyloid treatments aimed at the European market may need to show better cost-effectiveness to receive approval.
