Following Biogen’s submission of a Biologics License Application (BLA) to the FDA of aducanumab;

Alessio Brunello, Managing Pharma Analyst at GlobalData, a leading data and analytics company, offers his view:

“The potential approval for aducanumab is not likely to be the final treatment for Alzheimer’s disease (AD), but it could open doors for combination research, namely anti-Aβ therapies with anti tau-therapies. Combination therapy is likely the way to go for the Aβ inhibitor treatment class, but combination research cannot be explored until a drug is approved. The approval could also encourage companies with other amyloid-based therapies that failed before to continue testing and attract investors to raise money into the research.

“The data from the EMERGE study were positive with convincing clinical efficacy with the high-dose arm. However, the ENGAGE study did not show the same pattern of target engagement or clinical efficacy. Key opinion leaders (KOLs) interviewed by GlobalData expressed mixed opinion about the potential approval from the FDA as its standard of approval is substantial evidence of efficacy and the cumulative data for aducanumab are not really exhaustive to meet this standard. However, they are unlikely to turn down aducanumab even if its benefit is modest, given the lack of any therapy that is truly efficacious.

“GlobalData expected global sales of the drug to be high, equating to $4,191m of sales in 2028 in the eight major markets (8MM*), as there are no disease modifying drugs for this indication.

“KOLs interviewed by GlobalData were consistently enthusiastic about aducanumab’s potential and identified it as the most promising drug in the AD pipeline. However, its infusion route of administration could be an issue where infusion centers aren’t available and concerns regarding the developing of Amyloid Related Imaging Abnormalities (ARIA) can represent a barrier for the uptake for aducanumab. Physicians also will be obliged to do regular MRI to patients to see if they develop any ARIA reactions. This potentially could be a limitation, as repeated MRI could be also a limitation on a medical economic point of view.

“Experts in the field agreed that the drug actually works to clear amyloid plaques in the brain. Even though the FDA could decide to approve the treatment due to the significant unmet needs in this disease area, where no drug has been approved in the past 17 years, experts expressed doubts about its approval from federal regulators.”

*8MM = US, France, Germany, Italy, Spain, UK, Japan and China