Drug intelligence / Profile preview

c1 esterase inhibitor + danazol + tranexamic acid

Development stage
Unknown
Lead developer
CSL
Modality
Recombinant Proteins and Enzymes, Small Molecules
Administration
Intravenous, Subcutaneous, Oral
01

Overview

A combination therapy approach for hereditary angioedema (HAE) that includes three different medications: 1. **C1 esterase inhibitor**: A plasma-derived or recombinant protein that replaces the missing or dysfunctional C1 inhibitor in patients with HAE. It helps regulate the complement and contact system pathways, preventing spontaneous activation that leads to angioedema attacks. 2. **Danazol**: An attenuated androgen that increases hepatic synthesis of C1 inhibitor, helping to maintain higher levels of functional C1 inhibitor in the bloodstream. 3. **Tranexamic acid**: An antifibrinolytic agent that inhibits plasminogen activation, reducing the formation of plasmin which can trigger bradykinin production and subsequent angioedema attacks. This multi-modal approach targets HAE through different mechanisms: direct replacement of the deficient protein, stimulation of endogenous production, and inhibition of pathways that trigger attacks.

02

Targets

Factor XIaFXIIa (Coagulation Factor XIIa)C1S (Complement component 1s subcomponent serine protease)LBS (Plasminogen lysine-binding sites)C1R (Complement component 1r subcomponent)AR (Adrenergic receptors)KLKB1 (Plasma kallikrein)

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