The European Medicines Agency (EMA) has granted orphan designation to efdoralprin alfa (SAR447537, formerly known as INBRX-101), an investigational recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein, for the potential treatment of alpha-1 antitrypsin deficiency (AATD) related emphysema, a rare respiratory condition with great unmet medical need.

The EMA grants orphan designation to potential new medicines addressing rare, life-threatening or debilitating medical diseases or conditions that affect no more than five in 10,000 individuals in the EU.

Efdoralprin alfa demonstrated superiority to a standard of care plasma-derived therapy in adults with AATD when dosed every three weeks (Q3W) or every four weeks (Q4W), meeting all primary and key secondary endpoints in the global phase II ElevAATe study (clinical study identifier: NCT05856331).

The US Food and Drug Administration (FDA) previously granted both fast track and Orphan Drug Designation (ODD) to efdoralprin alfa for the treatment of AATD-related emphysema. Efdoralprin alfa is currently in clinical development, and its safety and efficacy have not been evaluated by any regulatory authority. Sanofi plans to present the data at a forthcoming medical meeting and engage with global regulatory authorities on the appropriate next steps.