Goebeler M, Bata-Csörgő Z, De Simone C, et al. - Findings demonstrate good tolerability of efgartigimod (an engineered Fc fragment that inhibits the activity of the neonatal Fc receptor) as a treatment option for pemphigus. Also, efgartigimod showed an early impact on disease activity and outcome parameters in pemphigus cases, and its further assessment as a therapy for pemphigus is supported.

• An open-label phase II adaptive trial included 34 patients with mild-to-moderate pemphigus vulgaris or foliaceus; sequential cohorts received efgartigimod dosed at 10 or 25 mg kg ⁻¹ intravenously with various dosing frequencies, as monotherapy or as add-on therapy to low-dose oral prednisone.

• Adverse events were mostly mild, and in patients receiving efgartigimod 10 mg kg ⁻¹ and in those receiving 25 mg kg ⁻¹ , adverse events occurred in 84% and 87%, respectively, with similar adverse event profiles between dose groups.

• A great reduction occurred in serum total IgG and anti-desmoglein autoantibodies and was correlated with improved Pemphigus Disease Area Index scores.

• Early disease control with efgartigimod, as monotherapy or combined with prednisone, was achieved in 90% of patients after a median of 17 days.

• Within 2–41 weeks, 64% of patients achieved complete clinical remission with optimized, prolonged therapy with efgartigimod in combination with a median dose of prednisone 0·26 mg kg ⁻¹ per day (range 0·06–0·48).