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efdoralprin alfa
- Development stage
- Phase 2
- Lead developer
- Sanofi
- Modality
- Fc-Fusion Proteins → Carrier/Scaffold Proteins → Recombinant Proteins and Enzymes
- Administration
- Intravenous
01
Overview
Efdoralprin alfa is a **recombinant, next-generation human alpha-1 antitrypsin (AAT)-Fc fusion protein** that contains two AAT molecules engineered for **extended serum half-life** and enhanced pharmacokinetics. It is being developed primarily for the treatment of **alpha-1 antitrypsin deficiency (AATD)**-associated emphysema and is designed to provide higher and more sustained serum levels of functional AAT than plasma-derived products. Efdoralprin alfa retains the physiological activity of wild-type AAT, specifically inhibiting neutrophil elastase (NE) and proteinase 3 (PR3), key proteases in the pathology of AATD, without significant off-target inhibition of thrombin or trypsin. The drug has received **Fast Track and Orphan Drug designations in the United States** and is currently in Phase 2 registrational trials.[1][3][7][8]
- Brand names
- efdoralprin alfa
- Other names
- efdoralprin alfa
02
Targets
ELANE (Human Neutrophil Elastase)PRTN3 (Proteinase 3)SERPINA1 (Alpha-1-antitrypsin)
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