By Fineline Cube 
Ultomiris (ravulizumab injection), a long‑acting C5 complement inhibitor under AstraZeneca’s (NYSE: AZN) Rare Disease business, was approved by China’s National Medical Products Administration (NMPA) for the treatment of adult and pediatric patients aged 1 month and older with paroxysmal nocturnal hemoglobinuria (PNH).
Regulatory Milestone
| Item | Detail |
|---|---|
| Agency | NMPA (China) |
| Product | Ultomiris (ravulizumab injection) |
| Class | Long‑acting C5 complement inhibitor |
| Indication | PNH in adult and pediatric patients aged ≥ 1 month |
| Approval Date | 20 Aug 2026 |
| Sponsor | AstraZeneca Rare Disease (NYSE: AZN) |
Clinical Evidence – Four Phase III Studies
The approval was based on comprehensive results from three global Phase III studies and one Chinese Phase III trial, demonstrating the efficacy and safety of ravulizumab in both adult and pediatric PNH patients:
| Study | Scope |
|---|---|
| ALXN1210‑PNH‑301 | Global Phase III |
| ALXN1210‑PNH‑302 | Global Phase III |
| ALXN1210‑PNH‑304 | Global Phase III |
| ALXN1210‑PNH‑323 | China Phase III |
Drug Profile & Mechanism of Action
- Mechanism: Long‑acting C5 complement inhibitor that blocks terminal complement‑mediated red blood cell destruction, the core driver of PNH
- Onset: Provides rapid complete C5 inhibition by the end of the first infusion
- Durability: Sustained complete C5 inhibition supports extended dosing intervals versus shorter‑acting complement therapies
- Approved Indications Globally: PNH, generalized myasthenia gravis (gMG), atypical hemolytic uremic syndrome (aHUS), and neuromyelitis optica spectrum disorder (NMOSD) across multiple countries and regions
Market Impact & Outlook
- China Rare‑Disease Access: The approval expands treatment options for Chinese PNH patients — including pediatric patients from 1 month of age — in a rare disease with historically limited therapeutic choices
- Dosing Advantage: Ultomiris’ long‑acting profile reduces infusion frequency, a meaningful quality‑of‑life and adherence benefit over short‑acting C5 inhibitors
- Portfolio Breadth: With approvals across four rare‑disease indications worldwide, ravulizumab anchors AstraZeneca’s Rare Disease franchise and strengthens its commercial position in China’s complement‑inhibitor market
- Competitive Landscape: The China entry of a long‑acting C5 inhibitor intensifies competition in the complement space, where factor B and other upstream mechanisms are also advancing through regulatory review
Forward‑Looking Statements
This brief contains forward‑looking statements regarding Ultomiris’ regulatory status, clinical profile, and commercial prospects in China. Actual results may differ due to risks including pricing and reimbursement negotiations, market adoption, and competitive dynamics in the complement inhibitor market.-Fineline Info & Tech