By Fineline Cube 
Jiangsu Hengrui Pharmaceuticals Co., Ltd. (SHA: 600276; HKG: 1276) announced that China’s National Medical Products Administration (NMPA) has approved its Category 1 drug HRS‑5965 for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have not previously been treated with complement inhibitors.
Regulatory Milestone
| Item | Detail |
|---|---|
| Agency | NMPA (China) |
| Approval Type | Category 1 innovative drug |
| Product | HRS‑5965 (complement factor B inhibitor) |
| Indication | Adult PNH patients naïve to complement inhibitor therapy |
| Approval Date | 21 Aug 2026 |
| Next Steps | Commercial launch and post‑marketing commitments; details not disclosed |
Drug Profile & Mechanism of Action
- Molecule: HRS‑5965, a complement factor B inhibitor developed in‑house by Hengrui
- Target: Complement factor B, a key driver of the alternative complement pathway implicated in PNH‑related hemolysis
- Positioning: Approved specifically for complement inhibitor‑naïve adult PNH patients, addressing an underserved segment of a rare and potentially life‑threatening hematologic disease
- Classification: Category 1 — the highest innovation tier under China’s drug review framework
Clinical Evidence – Phase III Trial (HRS‑5965‑301)
| Endpoint (Weeks 18–24) | Result (HRS‑5965) |
|---|---|
| Hemoglobin Response Rate (Hb increase ≥ 2 g/dL from baseline) | 95 % |
| Hemoglobin Normalization Rate (Hb ≥ 12 g/dL) | 70 % |
| Mean Hb Increase from Baseline | +5.5 g/dL |
The approval was based on HRS‑5965‑301, a multicenter, randomized, open‑label, active‑controlled Phase 3 trial. Comparator identity and additional secondary endpoints were not disclosed in the announcement.
Market Impact & Outlook
- PNH Landscape: PNH is a rare, potentially life‑threatening blood disorder in which uncontrolled complement activity destroys red blood cells, causing severe anemia, fatigue and thrombosis risk. The complement inhibitor space has historically been dominated by intravenous C5‑targeting therapies, with newer oral agents reshaping the competitive field.
- Hengrui’s Positioning: The approval gives Hengrui — one of China’s largest research‑driven drugmakers, dual‑listed in Shanghai (600276) and Hong Kong (1276) — an entry point in the rare‑disease complement space alongside its core oncology franchise.
- Differentiation Angle: An upstream factor B inhibitor approved for complement inhibitor‑naïve patients could appeal to physicians seeking hemoglobin normalization without prior exposure to other complement therapies; head‑to‑head comparisons versus incumbent agents remain undisclosed.
- Revenue Outlook: Commercial launch timing, pricing and sales forecasts were not disclosed.
Forward‑Looking Statements
This brief contains forward‑looking statements regarding regulatory developments, clinical outcomes, and commercial expectations for HRS‑5965. Actual results may differ due to risks including market adoption, competitive dynamics, and post‑marketing requirements.-Fineline Info & Tech