Sanofi efdoralprin alfa earns orphan designation in EU for alpha-1 antitrypsin deficiency related emphysema

Efdoralprin alfa (SAR447537, formerly known as INBRX-101) is a recombinant human AAT-Fc fusion protein being investigated in adults with AATD emphysema, with Q3W or Q4W dosing. The investigational restorative recombinant treatment is being studied to restore functional AAT levels to the normal range and inhibit neutrophil elastase, an enzyme that can cause lung tissue damage in patients with AATD. 

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 2 ElevAATe study (clinical study identifier: NCT05856331).

The US Food and Drug Administration (FDA) previously granted both fast track and orphan drug designation 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.