birtamimab

Birtamimab is a humanized monoclonal antibody designed to specifically and selectively target misfolded amyloid light chains (both kappa and lambda) that accumulate in organs and cause dysfunction in patients with amyloid light chain (AL) amyloidosis. By binding to a cryptic epitope exposed only on misfolded light chains, birtamimab neutralizes toxic soluble aggregates and promotes the clearance of insoluble amyloid deposits via phagocytosis. This mechanism is complementary to current standard-of-care therapies, which reduce new production of misfolded proteins but do not remove existing toxic aggregates or organ-deposited amyloid. Birtamimab is being developed by Prothena for the treatment of AL amyloidosis, particularly targeting patients with advanced (Mayo Stage IV) disease. It has shown a significant survival benefit in this population in post hoc analyses from clinical trials and has received orphan drug designation from both the FDA and EMA as well as Fast Track designation from the FDA[3][5][6][8].

Other names

NEOD001NEOD-001NEOD 001ELT1-01ELT-1-01ELT 1-01HU2A4HU-2A4HU 2A4