Sobi Japan and Asahi Kasei: Empaveli approved in Japan for C3 glomerulopathy and primary immune complex membranoproliferative glomerulonephritis

Approval and indication

Swedish Orphan Biovitrum Japan (Sobi Japan) and Asahi Kasei Therapeutics announced Japanese approval of Empaveli 1080 mg subcutaneous solution (pegcetacoplan) for C3 glomerulopathy and primary immune complex membranoproliferative glomerulonephritis. The approval includes patients aged 12 years and older, making Empaveli the first complement inhibitor in Japan authorised for these indications in pediatric patients.

Drug mechanism and prior status

Empaveli is a PEGylated peptide that binds complement proteins C3 and C3b to inhibit upstream complement activation. The therapy was previously approved in Japan in 2023 for paroxysmal nocturnal hemoglobinuria; Sobi Japan is the marketing authorisation holder and Asahi Kasei Therapeutics is the distributor.

Clinical evidence (VALIANT)

Approval was based on the global Phase III VALIANT trial, a randomized, placebo-controlled, double-blind, multicentre study in patients aged 12 and older that enrolled 124 patients. At Week 26 Empaveli reduced urine protein-to-creatinine ratio by 68.1% versus placebo, with renal function stabilisation shown as an eGFR difference of +6.3 mL/min/1.73 m² versus placebo. Kidney biopsy results showed a ≥2‑grade reduction in C3c staining in 74.3% of treated patients versus 11.8% with placebo. The safety profile aligned with previously established data for Empaveli.

Clinical and commercial plans

Sobi Japan and Asahi Kasei Therapeutics will engage nephrology, pediatric nephrology and pathology communities to raise awareness, support early diagnosis and promote appropriate use of Empaveli for these rare, progressive kidney diseases that have limited disease-specific therapies and may recur after transplantation.