Sanofi's Efdoralprin Alfa Gains EU Orphan Designation for AATD Emphysema
Sanofi’s investigational restorative recombinant therapy efdoralprin alfa has received EU orphan drug designation for alpha-1 antitrypsin deficiency–related emphysema, following positive phase II head-to-head results demonstrating success across all primary and key secondary endpoints versus the current plasma-derived standard of care.
Efdoralprin Alfa | 20/12/2025 | By News Bureau
Sanofi's Efdoralprin Alfa Meets Key Goals in Phase-II AATD Emphysema Study
Efdoralprin Alfa was previously granted fast track and Orphan Drug Designation (ODD) by the US Food and Drug Administration (FDA) for the treatment of AATD Emphysema. It is currently under clinical investigation, and its safety and efficacy have not been evaluated by any regulatory authority.
Efdoralprin Alfa | 27/10/2025 | By Dineshwori | 122
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