Sanofi’s Efdoralprin Alfa Phase 2 Study Results

Sanofi announced positive results from the global ElevAATe phase 2 study for efdoralprin alfa (SAR447537), an investigational recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein. The study met all primary and key secondary endpoints in adults with alpha-1 antitrypsin deficiency (AATD) emphysema. Efdoralprin alfa demonstrated superiority in a head-to-head study versus a standard of care plasma-derived therapy, showing a statistically significant greater mean increase in functional AAT levels within the normal range. The study also supported both three-week and four-week dosing regimens, potentially improving convenience compared to weekly plasma-derived therapy. Efdoralprin alfa was well tolerated with a similar adverse event profile to plasma-derived therapy. Sanofi plans to present the data at a forthcoming medical meeting and engage with global regulatory authorities on the next steps.