Sanofi has announced encouraging results from its Phase 2 ElevAATe clinical trial evaluating efdoralprin alfa (SAR447537), a recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein, for the treatment of emphysema caused by alpha-1 antitrypsin deficiency (AATD). The investigational therapy, as reported on drugs.com, met all primary and key secondary endpoints, demonstrating superior efficacy compared to standard weekly plasma-derived AAT augmentation therapy.

Administered every three or four weeks, efdoralprin alfa significantly increased functional AAT levels within the normal range, as measured by trough concentrations at steady state. Patients receiving the recombinant therapy also spent a higher percentage of time with protective protein levels above the lower limit of normal, a critical factor in managing AATD-related lung damage.

The treatment was well tolerated, with a safety profile comparable to existing plasma-derived options. Further safety data will be collected in the ongoing ElevAATe OLE extension study.

Experts in the field, including Dr. Igor Barjaktarevic of UCLA, highlighted the potential of efdoralprin alfa to offer a more convenient and effective alternative to current therapies, which rely on frequent infusions and blood donations. Sanofi’s Global Head of Development, Dr. Christopher Corsico, emphasized the importance of maintaining protective AAT levels and the company’s commitment to addressing unmet needs in rare respiratory diseases.

Efdoralprin alfa has received fast track and orphan drug designations from the U.S. FDA. While still under investigation, the therapy represents a potential shift in the treatment paradigm for AATD, a genetic condition affecting approximately 235,000 people globally—many of whom remain undiagnosed.

Sanofi plans to present the full data at an upcoming medical conference and engage with regulatory authorities to determine next steps.