Gefurulimab — Investigational Agent
Bispecific Antibody · Phase 3 · AstraZeneca / Alexion
Mechanism of Action
Subcutaneously administered bivalent, bispecific anti-C5 nanobody (VHH domain) that also binds serum albumin to extend half-life. Gefurulimab clamps complement C5 to prevent its cleavage into C5a and C5b, blocking assembly of the terminal membrane attack complex and the resulting intravascular hemolysis. Its small nanobody format enables low-volume subcutaneous self-administration.
Investigational Indications
- Generalized myasthenia gravis (lead Phase 3 indication)
- Paroxysmal nocturnal hemoglobinuria (PNH) — investigational interest as a convenient subcutaneous C5 inhibitor