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Sanofi Secures EU Orphan Designation For Efdoralprin Alfa In Alpha-1 Antitrypsin Deficiency Related Emphysema

Benzinga·12/17/2025 06:19:27
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Sanofi's efdoralprin alfa earns orphan designation in the EU for alpha-1 antitrypsin deficiency related emphysema

  • Additional orphan designation reinforces Sanofi's commitment to developing treatments for rare diseases
  • Efdoralprin alfa, an investigational restorative recombinant therapy, recently met all primary and key secondary endpoints in phase 2 head-to-head study versus a plasma-derived standard of care