Danicopan added to eculizumab led to clinical benefit in patients with paroxysmal nocturnal hemoglobinuria (PNH), based on phase 2 results published in Blood.

PNH is a rare disease that puts patients at risk of anemia requiring transfusions and thrombotic events. Eculizumab has greatly improved outcomes and quality of life for patients with PNH, but some patients may have residual anemia and remain transfusion dependent.

Danicopan is a complement factor D (FD) inhibitor. The study authors conducted a 24-week, phase 2 clinical trial of patients with PNH and inadequate response to eculizumab to determine if there’s a benefit to adding danicopan to eculizumab.

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A total of 11 patients were included in the 24-week efficacy analysis. All patients were on stable eculizumab therapy at the start of the trial.

The mean Hgb increased from baseline to week 24 (7.9 g/dL to 10.3 g/dL, P =.0001). Patients also had improvements in multiple PNH laboratory markers.

Before treatment with danicopan, 10 patients had received multiple transfusions. After starting treatment with danicopan, only 1 patient received a single transfusion. Patients also had improvements in fatigue levels.

Danicopan was well-tolerated, with the most common treatment-emergent adverse events (TEAEs) being cough, headache, nasopharyngitis, abdominal pain, arthralgia, fatigue, nausea, oropharyngeal pain, pain in extremity, and pain at vaccination site. Most TEAEs were mild to moderate, and 4 patients had severe TEAEs.

Overall, the results indicated that danicopan added to eculizumab can improve outcomes for patients who remain anemic and transfusion dependent.

Disclosure: One or more study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures.


Kulasekararaj AG, Risitano AM, Maciejewski JP, et al. Phase 2 study of danicopan in patients with paroxysmal nocturnal hemoglobinuria with an inadequate response to eculizumab. Blood. 2021;138(20):1928-1938. doi:10.1182/blood.2021011388