On 10 June, the Peripheral and Central Nervous System Drugs Advisory Committee convened to advise the US Food and Drug Administration (FDA) on the safety and efficacy of the drug donanemab from Eli Lilly, which is being considered for approval. During the meeting, advisors comprehensively reviewed the data of the TRAILBLAZER-ALZ 2 trial Phase 3 study, which evaluated the efficacy and safety of donanemab in early symptomatic Alzheimer's disease (AD) -MCI or mild dementia due to AD- with the presence of confirmed Alzheimer's disease neuropathology.
The trial enrolled 1,736 participants, across eight countries, selected based on cognitive assessments in conjunction with amyloid plaque imaging and tau staging by positron emission tomography (PET) imaging. In its discussion, the FDA had asked the panel to consider some unique aspects of Lilly's trial, especially that study participants stopped taking the drug as soon as their amyloid was cleared and the study's inclusion of participants based on tau levels. The 11 committee members voted unanimously in favour of the monoclonal antibody donanemab, being effective for people with an early stage of AD and that the drug’s benefits outweigh the risks.
The panel, however, raised concerns about certain safety risks and insufficient representation of underrepresented group like Hispanic and Black people in the Phase 3 trial. The FDA will soon decide on donanemab’s marketing authorisation. The FDA is not compelled to follow the recommendation of the independent panel of experts but it often does so.