Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab

Phase 2 Completed 12 enrolled Alexion Pharmaceuticals, Inc. Achillion, a wholly owned subsidiary of Alexion · collab

Non-randomizedSequentialOpen-labelTreatment

Summary

To determine the effectiveness of ACH-0144471 (also known as danicopan and ALXN2040) in improving anemia when given with eculizumab for 24 weeks in participants with PNH. Danicopan dose may be increased within each participant, to a maximum of 200 milligrams (mg) three times daily (TID) based on safety and efficacy at protocol-specified time points.

Timeline

Start: 2018-05-08
Primary completion: 2019-09-20
Completion: 2023-01-05

Publications

Kulasekararaj AG, Risitano AM, Maciejewski JP, Notaro R, Browett P, Lee JW, Huang M, Geffner M, Brodsky RA. Phase 2 study of danicopan in patients with paroxysmal nocturnal hemoglobinuria with an inadequate response to eculizumab. Blood. 2021 Nov 18;138(20):1928-1938. doi: 10.1182/blood.2021011388.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Danicopan	Small molecule	100 mg	Oral
Subject	Danicopan	Small molecule	150 mg	Oral
Subject	Danicopan	Small molecule	200 mg	Oral
Background	Eculizumab	Monoclonal antibody	—	Intravenous

Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab

Summary

Timeline

Publications

Drugs

Indications