Vemircopan — Investigational Agent
Other · Phase 2 · AstraZeneca / Alexion
Mechanism of Action
Oral small-molecule inhibitor of complement Factor D, the rate-limiting protease of the alternative complement pathway. By blocking Factor D, vemircopan prevents formation of the alternative-pathway C3 convertase (C3bBb), suppressing both C3 opsonin deposition on PNH red cells (the driver of extravascular hemolysis) and downstream C5-mediated intravascular hemolysis. As a proximal, orally bioavailable inhibitor it is being explored as a convenient monotherapy alternative to injectable C5 inhibitors.
Investigational Indications
- Paroxysmal nocturnal hemoglobinuria (PNH) — monotherapy in complement-inhibitor-naive and switch patients