SNY's Rare Disease Drug Efdoralprin Alfa Gets EMA's Orphan Drug Tag

Core Insights - Sanofi (SNY) has received Orphan Drug Designation (ODD) from the European Medicines Agency (EMA) for efdoralprin alfa, an investigational biologic drug aimed at treating alpha-1 antitrypsin deficiency (AATD) related emphysema in adults [1][8] Drug Development and Mechanism - Efdoralprin alfa is a recombinant human AAT protein designed to inhibit neutrophil elastases, which cause lung tissue damage in AATD patients [3] - The drug is currently in mid-stage development and was integrated into Sanofi's rare disease pipeline following the acquisition of Inhibrx in 2024 [4] Clinical Study Results - The ODD was granted based on positive results from the global phase II ElevAATe study, which showed that efdoralprin alfa administered every three weeks (Q3W) or four weeks (Q4W) significantly increased functional AAT levels compared to weekly plasma-derived therapy [7][8] - Treatment with efdoralprin alfa also resulted in a higher percentage of days with functional AAT levels above the normal range, meeting key secondary endpoints of the study [9] Market Performance - Over the past year, Sanofi's shares have increased by 1.3%, while the industry has seen a rise of 13.8% [6]