First oral once-daily complement Factor B inhibitor approved in China, now racing to file a U.S. IND — thesis turns on whether ex-China data can anchor an FDA path

Nuvectis is a clinical-stage company developing ciprocopan, an oral targeted therapy for paroxysmal nocturnal hemoglobinuria (PNH), which is approved in China but not yet approved in the United States.

Ciprocopan (NXP100) became the first globally approved oral once-daily complement Factor B inhibitor for PNH in July 2026, with Phase 3 data beating eculizumab 59.5% vs. 8.3% in treatment-naive patients. The bull case rests on whether that China-derived Phase 3 dataset, generated under a licensed ex-China structure, can support an FDA registration without a U.S. confirmatory trial. The principal risk is regulatory: single-arm Phase 3 data in C5-inhibitor-failed PNH and generalizability questions on the treatment-naive dataset could complicate FDA's acceptability of foreign-only evidence.