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**Zoracopan (ALXN2080)** is an investigational, orally administered **small molecule** that selectively inhibits **Complement Factor D (CFD)**, a serine protease essential for activation of the alternative pathway (AP) of the complement system. Developed by **Alexion Pharmaceuticals** (now part of **AstraZeneca Rare Disease** following the 2021 acquisition), it targets diseases driven by AP dysregulation, such as rare hematological, renal, and ophthalmological disorders. As a **New Molecular Entity**, Zoracopan has completed Phase 1 studies (2022-2024) demonstrating favorable safety, pharmacokinetics, and drug-drug interaction profiles with agents like rosuvastatin, metformin, itraconazole, fluconazole, and carbamazepine; it follows the precedent of Alexion/AstraZeneca's approved oral CFD inhibitor **danicopan (Voydeya)** for paroxysmal nocturnal hemoglobinuria (PNH).[1][3]
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