AstraZeneca (AZN.US) relizumab approved for marketing in China for the 3rd indication

Zhitong Finance App learned that on August 20, the official website of the National Drug Administration (NMPA) showed that AstraZeneca (AZN.US) “rilizumab” was approved as a new domestic indication for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Ralizumab was first approved for use in China in April 2025 to treat generalized myasthenia gravis in adults with positive anti-AChR antibodies, and was later extended to NMOSD. This is the 3rd indication that the drug has been approved domestically.

Ralizumab (ALXN-1210) is an important rare disease pipeline acquired by AstraZeneca after spending 39 billion US dollars to acquire Alexion. This is a long-acting C5 complement inhibitor. It was first approved in the US in 2018, and successively approved in the European Union and Japan in 2019. The trade name is Ultomiris.

Currently, overseas, the approved indications for this drug include paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (AHUS), myasthenia gravis (GMG), and optic neuromyelitis (NMOSD). In 2025, the drug's global sales reached US$4.718 billion, making it the best-selling product in AstraZeneca's rare disease segment.

AstraZeneca has formed an iterative product upgrade path in the field of complement inhibitors. The first approved product was the C5 inhibitor ecuzumab. Relizumab extended the administration interval from once every 2 weeks to once every 8 weeks, further improving the convenience of medication use.

According to the Insight database, 6 complement inhibitors have already been marketed in the domestic PNH sector, namely Roche's corvalimab, AstraZeneca's ecuzumab, Novartis's ipucopam, Lanocopam, Hisco's sipucopam, and Hengrui's riconcopam.

Ralizumab has now been approved as a new indication, making it the 7th domestic and 4th imported PNH treatment drug. Unlike other products, rilizumab provides a longer-lasting treatment option for domestic PNH patients with an 8-week dosing regimen.