News about "EU Orphan Designation "

Sanofi's Efdoralprin Alfa Gains EU Orphan Designation for AATD Emphysema

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.

EU Orphan Designation | 20/12/2025 | By News Bureau


 

 

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