AstraZeneca Strengthens China Rare-Disease Position With New Real-World PNH Study Update

AstraZeneca (AZN) announced an update on their ongoing clinical study.

AstraZeneca Updates Real-World Study on Rare Blood Disorder in China

The study, officially titled “A Real-world, Multi-center, Prospective, Observational Study for Paroxysmal Nocturnal Haemoglobinuria (PNH) in China,” aims to better understand how this rare blood disorder is treated and managed in everyday clinical practice. It focuses on describing how the disease progresses, how patients respond to current care, and what outcomes they experience, filling a major information gap in the Chinese market for this condition.

The key treatment in focus is eculizumab, a targeted drug that blocks part of the immune system that drives damage in PNH. It is not an experimental medicine in this study but a recently approved treatment in China, and the research tracks how it and other real-world medical approaches are being used and how patients fare over time.

The study is observational and non-randomized, meaning doctors continue to treat patients as they normally would, without any assignments by the sponsor. It uses a cohort model, following groups of PNH patients forward in time to see what happens under routine care. There is no blinding or placebo, and the main purpose is to collect practical, real-world information rather than test a new drug or dosing strategy.

The study began after submission on October 30, 2023, and followed patients prospectively for about one year, consistent with its planned target duration. It is listed as completed, indicating that follow-up and data collection are done. The most recent update, dated December 17, 2025, signals that the trial record and possibly the underlying analyses have been refreshed, which is relevant for investors tracking the maturity of AstraZeneca’s evidence base in PNH.

For investors, this update supports the long-term positioning of AstraZeneca’s complement inhibitor franchise in China, where PNH remains a niche but strategic market. Strong, real-world data can help drive broader physician confidence, support reimbursement discussions, and defend share against rivals in complement inhibition and rare hematology. While the direct revenue impact will be modest given the small patient population, a credible real-world dataset in China strengthens AstraZeneca’s overall rare disease profile and may influence sentiment positively, especially when compared with global competitors seeking to expand in similar ultra-rare indications.

The study has been completed and recently updated, with further information available on the ClinicalTrials portal.

To learn more about AZN’s potential, visit the AstraZeneca drug pipeline page.