By Fineline Cube 
CSPC Pharmaceutical Group Limited (HKG: 1093) announced that its independently developed Class 1 chemical novel drug SYH2061 Injection (a GalNAc‑conjugated double‑stranded small interfering RNA) has been granted clinical‑trial approval by the National Medical Products Administration (NMPA). The program targets IgA nephropathy and other complement‑mediated disorders, marking the first Chinese siRNA candidate to enter the clinical‑trial stage with an ultra‑long‑acting C5‑silencing profile.
Key Highlights
| Feature | Details |
|---|---|
| Drug | SYH2061 Injection – siRNA targeting complement protein C5 |
| Delivery | Subcutaneous, GalNAc‑conjugated for liver‑specific uptake |
| Indication | IgA nephropathy & other complement‑mediated diseases |
| Regulatory Status | NMPA clinical‑trial approval (Phase 1) – first independent Chinese siRNA in trial |
| Mechanism | Engineered siRNA with optimized sequence & chemical modifications for sustained gene silencing |
Why SYH2061 Matters
- First‑of‑its‑kind in China: The drug represents the first domestically‑developed siRNA to secure clinical‑trial approval, breaking a long‑standing dependence on imported RNA therapeutics.
- Ultra‑long‑acting C5 knockdown: Leveraging GalNAc‑mediated hepatocyte targeting, SYH2061 achieves sustained suppression of C5 protein, potentially reducing the need for frequent dosing.
- Complement‑mediated disease focus: By directly silencing a key driver of inflammation, SYH2061 offers a novel mechanism for conditions such as IgA nephropathy, atypical hemolytic‑uremic syndrome, and paroxysmal nocturnal hemoglobinuria.
Regulatory Pathway
- NMPA Clinical‑Trial Approval: The drug entered the “Class 1 chemical novel drug” pathway, a highly selective category reserved for advanced therapeutics.
- Expedited Review: CSPC benefited from the NMPA’s priority review framework, underscoring the unmet medical need in complement disorders.
- Next Steps: CSPC will launch a Phase 1/2 dose‑escalation study in early 2026, with plans to collaborate with academic centers for biomarker development.
Commercial Outlook
- Market Potential: IgA nephropathy affects > 150 million individuals worldwide; China alone accounts for ~ 10 % of the global patient population.
- Pricing Strategy: CSPC is negotiating with Chinese payers to secure a reimbursement pathway that reflects the drug’s disease‑modifying potential.
- Global Expansion: Successful clinical outcomes could pave the way for regulatory submissions in the U.S. (FDA) and EU (EMA), leveraging CSPC’s existing commercialization platform.
Forward‑Looking Statements
This release contains forward‑looking statements that involve risks and uncertainties. Actual results may differ materially.-Fineline Info & Tech