LB773 Treating pemphigus vulgaris (PV) and foliaceus (PF) by inhibiting the neonatal Fc receptor: Phase 2 multicentre open-label trial with efgartigimod

Abstract

Efgartigimod, an engineered Fc fragment that inhibits the activity of the neonatal Fc receptor (FcRn), was evaluated in an open-label phase 2 adaptive trial (NCT03334058). Thirty-four mild to moderate PV or PF patients were enrolled to evaluate the safety, pharmacodynamics, pharmacokinetics, and efficacy of efgartigimod. In four sequential cohorts, efgartigimod was dosed at 10 or 25 mg/kg intravenously with various dosing frequencies, as monotherapy or add-on therapy to low-dose oral prednisone.