After consulting with the FDA, Biogen has announced that it will pursue regulatory approval for aducanumab, an investigational treatment for early Alzheimer’s disease (AD).
The Phase 3 EMERGE Study met its primary endpoint showing a significant reduction in clinical decline, and Biogen believes that results from a subset of patients in the Phase 3 ENGAGE Study who received sufficient exposure to high dose aducanumab support the findings from EMERGE.
The decision to file is based on a new analysis, conducted by Biogen in consultation with the FDA, of a larger dataset from the Phase 3 clinical studies that were discontinued in March 2019 following a futility analysis.
This new analysis of a larger dataset that includes additional data that became available after the pre-specified futility analysis shows that aducanumab is pharmacologically and clinically active as determined by dose-dependent effects in reducing brain amyloid and in reducing clinical decline as assessed by the pre-specified primary endpoint Clinical Dementia Rating-Sum of Boxes (CDR-SB).
In both studies, the safety and tolerability profile of aducanumab was consistent with prior studies of aducanumab.
Michel Vounatsos, CEO of Biogen, said: “We are hopeful about the prospect of offering patients the first therapy to reduce the clinical decline of Alzheimer’s disease and the potential implication of these results for similar approaches targeting amyloid beta.”
Based on discussions with the FDA, the company plans to file a Biologics License Application (BLA) in early 2020 and will continue dialogue with regulatory authorities in international markets including Europe and Japan.
The BLA submission will include data from the Phase 1/1b studies as well as the complete set of data from the Phase 3 studies.