Astrazeneca PLC Sponsored ADR (AZN.US) has received approval for the third indication of Repatha in China.

On August 20, the official website of the National Medical Products Administration (NMPA) announced that Astrazeneca PLC Sponsored ADR (AZN.US) has received approval for a new indication for "Ravulizumab" in China, which is used for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Ravulizumab was first approved for entry into China in April 2025 for the treatment of adult patients with anti-AChR antibody-positive generalized myasthenia gravis, and later expanded its indication to NMOSD. This marks the third indication approved for the drug in the domestic market. Ravulizumab (ALXN-1210) is an important rare disease pipeline obtained by Astrazeneca PLC Sponsored ADR following its significant $39 billion acquisition of Alexion. It is a long-acting C5 complement inhibitor that was first approved in the United States in 2018 and subsequently approved in the European Union and Japan in 2019 under the brand name Ultomiris. Currently, overseas, the drug has been approved for several indications including paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), myasthenia gravis (gMG), and neuromyelitis optica spectrum disorder (NMOSD). In 2025, the global sales of this drug reached $4.718 billion, making it the best-selling product in the rare disease sector of Astrazeneca PLC Sponsored ADR. Astrazeneca PLC Sponsored ADR has established a product iteration and upgrade path in the complement inhibitor field, with the first approved product being the C5 inhibitor Eculizumab. Ravulizumab extends the dosing interval from every 2 weeks to once every 8 weeks, further enhancing the convenience of treatment. According to the Insight database, there were already 6 complement inhibitors approved in the domestic PNH field, namely Roche's Cevulizumab, Astrazeneca PLC Sponsored ADR's Eculizumab, Novartis AG Sponsored ADR's Iptacopan, Langlai Technology's Lanadelumab, Haisco Pharmaceutical Group's Siponimod, and Heng Rui's Licocadraw. With the approval of this new indication, Ravulizumab has become the seventh PNH treatment drug in China and the fourth imported product. Unlike other products, the once-every-8-weeks dosing regimen of Ravulizumab provides a longer-lasting treatment option for PNH patients in China.