After a longer-than-expected wait, Biogen completed an FDA filing for its controversial Alzheimer’s drug aducanumab, the company announced Wednesday.
Biogen surprised the Alzheimer’s disease community last October when it announced plans to pursue regulatory approval for aducanumab, an investigational monoclonal antibody targeting beta-amyloid protein in early Alzheimer’s disease, months after the company and its partner Eisai had terminated phase III trials of aducanumab that failed futility analyses.
The parallel EMERGE and ENGAGE trials of early-stage and mild Alzheimer’s were terminated in March 2019 when an interim analysis determined aducanumab was unlikely to outperform placebo at completion. But in October, the company said a review of previously unavailable data showed the drug actually reduced cognitive decline in EMERGE, but not in ENGAGE.
At the Clinical Trials on Alzheimer’s Disease (CTAD) meeting in San Diego in December, Biogen vice president Samantha Budd Haeberlein, PhD, indicated that different enrollment periods and a midstream protocol amendment accounted for differences in EMERGE and ENGAGE data, pointing to a subgroup of patients who received 14 treatments of the highest dose of aducanumab who reaped the most benefit.
Experts attending CTAD were hesitant to draw conclusions, however, especially from subsets of data.
“There are biomarker signals and clinical signals that show promise,” Howard Fillit, MD, founding executive director of the Alzheimer’s Drug Discovery Foundation in New York City, said at the time. “But to get this drug over the regulatory hurdle of an FDA approval, or just to convince the scientific community that it really works, we need to see another phase III trial.”
“This drug may potentially have a huge impact, in terms of societal cost and in terms of patients’ ability to pay co-pays over a long period of time,” Fillit told MedPage Today. “We’re talking potentially millions of people on a relatively expensive drug on a monthly basis. It’s going to be a very big payer decision.”
The FDA now has up to 60 days to decide whether to accept Biogen’s application for review. If accepted, Biogen expects the FDA will inform the company whether the drug has been granted priority review designation.
“The aducanumab [biologics license application] is the first filing for FDA approval of a treatment that addresses the clinical decline associated with this devastating condition, as well as the pathology of the disease,” Biogen CEO Michel Vounatsos said in a statement. “We are committed to driving progress for the Alzheimer’s disease community and look forward to the FDA’s review of our filing.”