SNY's Efdoralprin Alfa Meets Key Endpoints in Rare Disease Study

Core Insights - Sanofi's phase II ElevAATe study for efdoralprin alfa in treating alpha-1 antitrypsin deficiency (AATD) emphysema met all primary and key secondary endpoints [1][8][10] - Efdoralprin alfa demonstrated a statistically significant increase in functional AAT levels compared to weekly plasma-derived therapy [2][9] - The dosing regimens of every three weeks (Q3W) and four weeks (Q4W) may enhance convenience for patients [9][10] Study Results - The ElevAATe study showed that efdoralprin alfa resulted in a greater mean increase in average functional AAT concentrations and a higher percentage of days with levels above the lower limit of normal [3][8] - The treatment's efficacy was confirmed by achieving higher functional AAT levels compared to the standard weekly therapy [8][10] Safety and Future Development - Additional safety follow-up for efdoralprin alfa will be assessed in the phase II ElevAATe OLE study [3] - The FDA has granted fast track and orphan drug designations for efdoralprin alfa, indicating its potential significance in treating AATD emphysema [10] Market Performance - Year-to-date, Sanofi's shares have increased by 3%, while the industry has risen by 6% [6]