Ciprocopan Approved in China for Treatment-Naive Paroxysmal Nocturnal Hemoglobinuria – Nuvectis Pharma / Haisco
Nuvectis Pharma announced that ciprocopan (NXP100), a once-daily oral complement Factor B inhibitor, received marketing approval from the National Medicinal Products Administration of China (NMPA) for the treatment of patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) previously untreated with Complement inhibitors (treatment naive).
As the world’s first approved once-daily oral Complement Factor B inhibitor, ciprocopan offers patients living with PNH a new, effective, safe and convenient treatment option and provides important clinical and regulatory validation and further supports the therapeutic potential of once-daily oral Factor B inhibition in PNH and other complement mediated diseases.
“We are delighted that ciprocopan received its first marketing approval in China for the treatment of patients with PNH previously untreated with Complement inhibitors, and congratulate our partner Haisco on this tremendous achievement.” – Ron Bentsur, Chairman and Chief Executive Officer of Nuvectis.
“With Factor B inhibitors expected to become the leading class in the multi-billion-dollar PNH market over time, ciprocopan, as the currently only once a day oral therapy, is well positioned to obtain meaningful market share, if approved outside of China. Beyond PNH, ciprocopan could become an important treatment option across several other complement-mediated diseases, where the convenience advantage could be important for patient compliance and long-term clinical outcomes.” – Ron Bentsur, Chairman and Chief Executive Officer of Nuvectis.
“This first ciprocopan approval is an important milestone for Chinese patients living with PNH and we are excited to work closely with Nuvectis in order to bring this important new treatment option to patients worldwide.” – Dr. Pangke Yan, Chief Executive Officer of Haisco.
The key basis for approval was a head-to-head Phase 3 study of ciprocopan versus Soliris (eculizumab) in patients with PNH patients previously untreated with Complement inhibitors. The study met all primary and secondary endpoints and demonstrated ciprocopan’s superiority over eculizumab, including reaching a hemoglobin target of 12 g/dL in 59.5% vs 8.3% in patients treated with eculizumab, a hemoglobin increase from baseline of approximately 5.0 g/dL vs 2.2 g/dL, and reduced transfusion requirements with 94.6% of patients vs 69.4% not requiring transfusions during the study. The study also demonstrated a favorable safety profile for ciprocopan vs eculizumab with no adverse events leading to treatment discontinuation or study withdrawal.
Unlike terminal complement inhibitors such as the C5 inhibitors Soliris and Ultromiris, Factor B inhibition blocks the amplification of the alternative pathway while preserving the classical and lectin pathways, designed to provide comprehensive control of both intravascular and extravascular hemolysis, leading to better efficacy compared to the C5 inhibitors’, as demonstrated in head to head clinical trials.
Haisco independently developed ciprocopan and is responsible for its development, regulatory approvals, and commercialization in China. Under the June 2026 exclusive license agreement with Haisco, Nuvectis holds exclusive rights to develop and commercialize ciprocopan outside Greater China, India and certain Southeast Asia countries.
