The European Medicines Agency (EMA) has granted orphan designation to biopharma major Sanofi's efdoralprin alfa, an investigational therapy for alpha-1 antitrypsin deficiency (AATD) related emphysema, a rare and serious respiratory condition with significant unmet medical need.

 

The EMA awards orphan designation to potential treatments for rare, life-threatening, or debilitating conditions affecting no more than 5 in 10,000 individuals in the EU.

 

Efdoralprin alfa, a recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein, showed superior efficacy to standard 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 2 ElevAATe study.

 

The US Food and Drug Administration (FDA) previously granted the therapy both fast track and orphan drug designations for AATD-related emphysema. Sanofi emphasized that efdoralprin alfa is still in clinical development, and “its safety and efficacy have not been evaluated by any regulatory authority.” The company plans to present the data at an upcoming medical meeting and engage with global regulators on next steps.

 

Efdoralprin alfa is designed to restore functional AAT levels to the normal range and inhibit neutrophil elastase, an enzyme that can damage lung tissue in patients with AATD. The therapy is being investigated in adults with AATD emphysema using Q3W or Q4W dosing. In addition to the EMA orphan designation, it has received FDA fast track and orphan drug status.