By Fineline Cube 
China Medical System Holdings (CMS; HKG: 0867) announced that the National Medical Products Administration (NMPA) has approved a clinical trial for CMS-D017, a novel selective small‑molecule inhibitor of complement factor B, in healthy Chinese participants. The drug will be evaluated for safety, tolerability, pharmacokinetics, and pharmacodynamics as a potential treatment for paroxysmal nocturnal hemoglobinuria (PNH) and other complement‑mediated diseases.
Regulatory Milestone & Drug Profile
| Item | Detail |
|---|---|
| Company | China Medical System Holdings (CMS; HKEX: 867) |
| Drug | CMS-D017 |
| Mechanism | Selective small‑molecule inhibitor of complement factor B |
| Indication | PNH (initial); future potential in kidney disease, AMD, myasthenia gravis |
| Approval | NMPA clinical trial authorization |
| Trial Design | Phase I in healthy Chinese participants |
| Preclinical Data | Excellent efficacy and safety demonstrated |
Market Impact & Commercial Outlook
- PNH Market: China PNH market valued at ¥500 million : (~US$70 million) in 2025, projected to reach ¥1.2 billion : by 2030 driven by novel complement inhibitors
- Complement‑Mediated Diseases: Represents $3 billion : global market opportunity across PNH, aHUS, AMD, and neuromuscular disorders
- Revenue Potential: Analysts project ¥2–4 billion (US$280–560 million) peak sales for CMS-D017 if approved across multiple indications
- Competitive Landscape: Positions CMS against Alexion (AstraZeneca) and Apellis; oral small‑molecule profile offers dosing convenience vs. injectable biologics
- Strategic Value: First NMPA‑approved clinical trial for a complement factor B inhibitor in China; validates CMS’s innovation strategy
- Next Steps: Phase I trial initiation expected Q2 2026; PNH patient enrollment targeted for H2 2026
Forward‑Looking Statements
This brief contains forward‑looking statements regarding clinical development timelines, regulatory pathways, and revenue projections for CMS-D017. Actual results may differ due to clinical trial outcomes, competitive dynamics, and NMPA review processes.-Fineline Info & Tech