Danicopan

Danicopan, sold under the brand name Voydeya, is a medication used for the treatment of paroxysmal nocturnal hemoglobinuria. It is a complement inhibitor which reversibly binds to factor D to prevent alternative pathway-mediated hemolysis and deposition of complement C3 proteins on red blood cells.

Danicopan was approved for medical use in Japan in January 2024, and in the United States in March 2024.

Medical uses
Danicopan is indicated as add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria.

Legal status
In February 2024, the Committee for Medicinal Products for Human Use of the EMA adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Voydeya, intended as add-on therapy to ravulizumab or eculizumab for the treatment of residual hemolytic anemia in adults with paroxysmal nocturnal hemoglobinuria (PNH). The applicant for this medicinal product is Alexion Europe.

Names
Danicopan is the international nonproprietary name.