FORT LEE, N.J., July 23, 2026
Nuvectis Pharma, Inc. announced that ciprocopan (NXP100) has received marketing approval from China’s National Medical Products Administration (NMPA) for the treatment of patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have not previously received complement inhibitor therapy. The approval marks the world’s first regulatory authorization for a once-daily oral Complement Factor B inhibitor, introducing a new treatment option for patients living with the rare blood disorder. The milestone is based on strong Phase 3 clinical data demonstrating superior efficacy and a favorable safety profile compared with Soliris (eculizumab). The approval also validates the therapeutic potential of oral Factor B inhibition and strengthens ciprocopan’s position as a promising therapy for PNH and other complement-mediated diseases requiring long-term treatment.
Phase 3 Trial Demonstrates Superior Efficacy Over Soliris
The marketing approval was primarily supported by a head-to-head Phase 3 clinical trial comparing ciprocopan with eculizumab (Soliris) in treatment-naïve PNH patients. The study successfully achieved all primary and secondary endpoints, demonstrating clear clinical advantages for ciprocopan. Approximately 59.5% of patients receiving ciprocopan achieved hemoglobin levels of at least 12 g/dL, compared with only 8.3% in the Soliris arm. Patients treated with ciprocopan also experienced an average hemoglobin increase of approximately 5.0 g/dL, versus 2.2 g/dL with eculizumab. Additionally, 94.6% of ciprocopan-treated patients remained transfusion-free, significantly outperforming the 69.4% reported for Soliris. Importantly, the study reported no adverse events leading to treatment discontinuation or study withdrawal, highlighting the therapy’s favorable safety profile alongside its improved clinical efficacy.
Novel Factor B Inhibition Offers Convenient Once-Daily Therapy
Unlike traditional C5 complement inhibitors such as Soliris and Ultomiris, ciprocopan targets Complement Factor B, blocking amplification of the alternative complement pathway while preserving the classical and lectin pathways. This differentiated mechanism is designed to provide more comprehensive control of both intravascular and extravascular hemolysis, addressing key limitations associated with currently available therapies. As the first once-daily oral Factor B inhibitor approved globally, ciprocopan eliminates the need for intravenous infusions, potentially improving patient convenience, long-term treatment adherence, and quality of life for individuals requiring lifelong therapy. Company executives believe the convenience of an oral treatment, combined with superior efficacy, positions ciprocopan to become a significant competitor within the rapidly expanding multi-billion-dollar PNH treatment market.
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Global Development Plans Extend Beyond China
The newly approved therapy was developed by Haisco, which is responsible for clinical development, regulatory approvals, and commercialization within China. Under an exclusive licensing agreement signed in June 2026, Nuvectis Pharma obtained exclusive rights to develop and commercialize ciprocopan outside Greater China, India, and selected Southeast Asian countries. In addition to the newly approved indication for treatment-naïve patients, a second marketing application covering PNH patients previously treated with anti-C5 therapies remains under regulatory review in China. Beyond PNH, both companies believe ciprocopan has broad therapeutic potential across multiple complement-mediated diseases, where its once-daily oral administration may improve long-term compliance and clinical outcomes. The approval represents a major milestone for Nuvectis Pharma, providing important regulatory validation of its complement-focused development strategy while laying the foundation for future global commercialization opportunities in rare hematologic and immune-mediated disorders.
Source: Nuvectis Pharma press release
