Alexion Pharmaceuticals, Inc. announced that VOYDEYA (danicopan) has been approved in the US as add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH). VOYDEYA is a first-in-class, oral, Factor D inhibitor developed as an add-on to standard-of-care ULTOMIRIS (ravulizumab-cwvz) or SOLIRIS (eculizumab) to address the needs of the approximately 10-20% of patients with PNH who experience clinically significant EVH while treated with a C5 inhibitor. The approval by the US Food and Drug Administration (FDA) was based on positive results from the pivotal ALPHA Phase III trial.

Results from the 12-week primary evaluation period of the trial were published in The Lancet Haematology. The ALPHA Phase III trial evaluated the efficacy and safety of VOYDEYA as add-on to ULTOMIRIS or SOLIRIS in patients with PNH who experienced clinically significant EVH.Results showed thatVOYDEYA met the primary endpoint of change in hemoglobin from baseline to week 12 and all key secondary endpoints, including transfusion avoidance and change in Functional Assessment of Chronic Illness Therapy ? Fatigue (FACIT-Fatigue) score.

Results from the ALPHA Phase III trial showed VOYDEYAwas generally well tolerated, and no new safety concerns were identified. In the trial, the most common treatment-emergent adverse events were headache, nausea, arthralgia and diarrhea.