On August 20, the National Medical Products Administration (NMPA) website showed that AstraZeneca PLC (NASDAQ: AZN) has received approval for a new indication for its drug "Ravulizumab" in China, specifically for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH).

Ravulizumab first entered the Chinese market in April 2025, initially approved for treating adult patients with generalized Myasthenia Gravis (gMG) who are positive for anti-AChR antibodies. Its indications were subsequently expanded to include Neuromyelitis Optica Spectrum Disorder (NMOSD). This latest approval marks the third indication for the drug within the country.

Ravulizumab (ALXN-1210) is a key asset in the rare disease portfolio that AstraZeneca PLC acquired through its $39 billion purchase of Alexion. As a long-acting C5 complement inhibitor, it was first approved in the United States in 2018, followed by approvals in Europe and Japan in 2019, where it is marketed under the brand name Ultomiris. Currently, outside of China, the drug's approved indications include PNH, atypical Hemolytic Uremic Syndrome (aHUS), gMG, and NMOSD.

In 2025, global sales for this medication reached $4.718 billion, making it the top-selling product in AstraZeneca PLC's rare disease segment. The company has established a clear product evolution path within the complement inhibitor space. Its first approved product in this category was the C5 inhibitor Eculizumab, and Ravulizumab extends the dosing interval from every two weeks to every eight weeks, significantly improving patient convenience.

According to data from the Insight database, six complement inhibitors were already available in China for PNH prior to this approval. These include Roche's Crovalimab, AstraZeneca PLC's own Eculizumab, Novartis's Iptacopan, Langlai Technology's Lanrocopan, Haisco Pharmaceutical's Siprocopan, and Hengrui's Licopan. With this new indication, Ravulizumab becomes the seventh PNH treatment overall and the fourth imported option available to patients in China.

What sets Ravulizumab apart from the other products is its once-every-eight-weeks dosing schedule, offering domestic PNH patients a more long-acting therapeutic alternative.