Globenewswire·2025-12-17 06:00Core Insights - The European Medicines Agency (EMA) has granted orphan designation to efdoralprin alfa for the treatment of alpha-1 antitrypsin deficiency (AATD) related emphysema, addressing a significant unmet medical need in a rare respiratory condition [1][2][3] Group 1: Efdoralprin Alfa Overview - Efdoralprin alfa (SAR447537, formerly INBRX-101) is an investigational recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein aimed at treating AATD emphysema [1][4] - The drug has shown superiority over standard plasma-derived therapy in adults with AATD in the global phase 2 ElevAATe study, meeting all primary and key secondary endpoints [2][4] - Efdoralprin alfa has also received fast track and orphan drug designations from the US FDA, indicating its potential significance in treating AATD [3][4] Group 2: Alpha-1 Antitrypsin Deficiency (AATD) - AATD is a rare inherited disorder characterized by low or absent levels of AAT, leading to progressive lung and liver tissue damage [5] - Approximately 235,000 individuals globally are affected by AATD, with nearly 100,000 in the US, and about 90% of these cases remain undiagnosed [5] Group 3: Sanofi's Commitment - Sanofi is dedicated to developing treatments for rare diseases, as evidenced by the recent orphan designation for efdoralprin alfa [7] - The company emphasizes its commitment to improving lives through innovative research and development in the biopharma sector [6]