AstraZeneca Advances PNH Treatment with Danicopan Study Update

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

Study Overview: AstraZeneca, in collaboration with Alexion Pharmaceuticals, is conducting a Phase 3 open-label study titled A Phase 3 Open-Label Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Significant Extravascular Hemolysis. The study aims to evaluate the efficacy of danicopan as an add-on treatment to ravulizumab or eculizumab by assessing hemoglobin change from baseline at Week 12 in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH) and clinically significant extravascular hemolysis (CS-EVH).

Intervention/Treatment: The intervention being tested is danicopan, a drug administered on a weight-based dosing regimen. It is intended to enhance the treatment of PNH by addressing extravascular hemolysis when used alongside ravulizumab or eculizumab.

Study Design: This interventional study is non-randomized with a single-group assignment. It is open-label, meaning no masking is involved, and its primary purpose is treatment. Participants will undergo a 12-week weight-based open-label treatment period, with the possibility of an extension for up to one year.

Study Timeline: The study began on June 3, 2024, with its primary completion and estimated completion dates yet to be announced. The latest update was submitted on October 27, 2025, indicating ongoing recruitment and progress.

Market Implications: This study update could positively influence AstraZeneca’s stock performance by showcasing their commitment to advancing treatments for rare diseases like PNH. Successful results may enhance investor confidence and position AstraZeneca favorably against competitors in the rare disease treatment market.

The study is ongoing, with further details available on the ClinicalTrials portal.