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gefurulimab
- Development stage
- Phase 1
- Lead developer
- Alexion Pharmaceuticals
- Modality
- Bispecific Antibodies → Multispecific Antibodies → Engineered Antibody Formats → Antibody-Based Therapeutics, Fc-Fusion Proteins → Carrier/Scaffold Proteins → Recombinant Proteins and Enzymes, Nanobodies (VHH) → Antibody Fragments → Engineered Antibody Formats → Antibody-Based Therapeutics, Monoclonal Antibodies → Antibody-Based Therapeutics
- Administration
- Subcutaneous
01
Overview
Gefurulimab is a humanized bispecific single-domain antibody (VHH) that targets complement component 5 (C5) and albumin. By binding to C5, gefurulimab blocks its cleavage into C5a and C5b, thereby inhibiting the terminal complement pathway, which reduces complement-mediated inflammation and cell lysis. The additional binding to albumin extends the drug’s half-life in circulation, allowing for convenient subcutaneous dosing. Gefurulimab is being developed primarily for generalized myasthenia gravis (gMG), an autoimmune neuromuscular disease characterized by muscle weakness and fatigue due to autoantibody-driven complement activation at the neuromuscular junction. The drug is currently in phase 3 clinical trials for gMG and has also entered early-phase studies for proteinuria[1][2][4][5][6][7].
- Other names
- gefurulimab
02
Targets
C5
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