Sanofi(US:SNY) Globenewswire·2025-10-22 05:00Core Insights - Sanofi's efdoralprin alfa has shown positive results in the ElevAATe phase 2 study for treating alpha-1 antitrypsin deficiency (AATD) emphysema, meeting all primary and key secondary endpoints [1][3][7] - The investigational drug demonstrated a statistically significant increase in functional AAT levels compared to standard weekly plasma-derived therapy [1][5] - Efdoralprin alfa is positioned as a potential first restorative recombinant therapy for AATD, offering less frequent dosing options [3][5] Study Results - The ElevAATe phase 2 study involved 97 patients, comparing efdoralprin alfa administered every three weeks or four weeks against weekly plasma-derived therapy [7] - Primary endpoint achieved: mean change in average functional AAT concentrations at steady state was significantly higher for efdoralprin alfa [1][7] - Key secondary endpoints also met, showing superior mean increase in fAAT average concentration and higher percentage of days above the lower limit of normal [1][7] Safety Profile - Efdoralprin alfa was well tolerated, exhibiting a similar adverse event profile to existing plasma-derived therapies [2] - Further safety assessments will be conducted in the ongoing ElevAATe OLE phase 2 study [2] Implications for Treatment - The results indicate a potential shift in AATD management, allowing patients to maintain normal AAT levels with less frequent dosing and independence from blood donation programs [3][4] - Current treatments require weekly infusions, leaving patients with fluctuating AAT levels, which efdoralprin alfa aims to address [3][4] Regulatory Status - Efdoralprin alfa has received fast track and orphan drug designations from the FDA for AATD emphysema treatment [3][6] - Sanofi plans to present the study data at a medical meeting and engage with regulatory authorities for next steps [3]