Biogen's Alzheimer’s Drug Aduhelm Faces Likely Rejection in Europe Amid Regulatory and Commercial Challenges

Compiled by Fan Dongdong

Recently, there is news indicating that European regulatory authorities do not intend to repeat the mistakes made by the U.S. FDA, avoiding the controversial situation caused by approving Biogen and Eisai's Alzheimer's drug Aduhelm.

Biogen announced that it has received a "negative trend vote" from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) regarding the application for Aduhelm. For Biogen, as one analyst noted, if the regulatory outcome for Aduhelm in Europe falls short of expectations, 40% of the drug’s potential revenue could be at risk. During the review process, the trend vote directly influences the opinions of CHMP expert members on the drug application. According to the European EMA guidelines, although the trend vote only records the informal positions of the reviewing experts, unless significant new information is provided, the final formal voting result may not change.

Preliminary Negative Opinion Indicates European Drug Reviewers Believe Aduhelm Should Not Be Approved for Alzheimer’s TreatmentA preliminary negative opinion means that European drug reviewers believe Aduhelm should not be approved for the treatment of Alzheimer's disease. This decision follows last week's oral hearing and a formal ruling is expected at the next European CHMP meeting in December this year. Beyond the EU market, Biogen and Eisai are also actively seeking breakthroughs in the Asian market. Japanese regulators may make a decision by the end of this year based on documents submitted in December 2020.

RBC Capital Markets analyst Brian Abrahams wrote in a Wednesday report that regulatory setbacks in the European market would cut 40% of Aduhelm's future potential revenue. Biogen Inc. also said in a statement that it would continue to maintain close communication and engagement with the European EMA and CHMP regarding the next steps for Aduhelm in Europe.

Abrahams noted that Biogen can appeal against the recommendation of the European CHMP, but the success rate of such a strategy has historically been low, at only about 20%. Given the existing dataset for the drug and the controversies surrounding it since its launch in the U.S., Abrahams predicts that Aduhelm will ultimately not pass through European regulatory authorities. Abrahams added that even if approved, reimbursement for the drug may be challenging given its high price. Aduhelm costs approximately $56,000 per year in the U.S., and since receiving FDA approval, the drug has caused a stir domestically due to its lack of clear efficacy and high pricing.

Since the drug was unexpectedly approved by the U.S. FDA in June, Biogen has been going through a tough time. Echoing the overwhelmingly negative opinions from the external FDA advisory committee, clinicians have also generally been reserved about adopting the drug. According to data, Aduhelm’s sales in the third quarter of this year were only $300,000.

However, Biogen insists that the drug has clear efficacy. Last week, at the Alzheimer's Disease Clinical Trials Conference, the company released more biomarker data on Aduhelm. The results showed that this anti-amyloid-beta drug can also reduce blood levels of phosphorylated tau, and amyloid plaques and tau tangles are two hallmarks of Alzheimer’s disease. Although the company described a "correlation" between the reduction in p-tau and cognitive improvement, the actual correlation value was very low.

In addition, the drug has also faced repeated setbacks in cooperation with health insurance. In August this year, the Department of Veterans Affairs decided to exclude Aduhelm from its formulary. The Centers for Medicare and Medicaid Services (CMS) is expected to rule early next year on how to cover Aduhelm under Medicare.

Besides Aduhelm's high pricing and uncertain efficacy, the drug even faces safety risks. According to a recent update from the FDA Adverse Event Reporting System in the United States, a patient died after receiving treatment with the drug, further fueling doubts about Aduhelm. A 75-year-old woman in Canada passed away after being diagnosed with brain swelling or amyloid-related imaging abnormalities, which are known side effects of Aduhelm.

Reference Source: Biogen’s Aduhelm, Already Battling US Skepticism, Faces Likely European Rejection