Voydeya (danicopan) is approved in Japan for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). It is indicated in Japan in combination with C5 inhibitor therapy when patients have had an insufficient response to such C5 inhibitors. Voydeya is a first-in-class, oral, Factor D inhibitor developed as add-on to proven standard-of-care Ultomiris or Soliris to address the needs of the subset of patients (approximately 10-20%) with PNH who experience clinically significant extravascular hemolysis (EVH) while treated with a C5 inhibitor.
The approval by the Japanese Ministry of Health, Labor and Welfare (MHLW) 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.
PNH is a rare and severe blood disorder characterized by the destruction of red blood cells within blood vessels, known as intravascular hemolysis (IVH), and white blood cell and platelet activation that can cause thrombosis (blood clots) and result in organ damage and potentially premature death.4-6 Immediate, complete and sustained terminal complement inhibition by blocking the C5 protein helps reduce symptoms and complications, resulting in improved survival for patients with PNH.6-9 Approximately 10-20% of people living with PNH who are treated with a C5 inhibitor experience clinically significant EVH, which can result in continued symptoms of anemia and require blood transfusions.
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, defined as hemoglobin ≤9.5 g/dL and absolute reticulocyte count levels ≥120x109/L. Results showed that Voydeya 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 Voydeya was 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 diarrhoea.
Voydeya has been granted Breakthrough Therapy designation by the US Food and Drug Administration and PRIority MEdicines (PRIME) status by the European Medicines Agency. Voydeya has also been granted Orphan Drug Designation in the US, EU and Japan for the treatment of PNH. Regulatory submissions for Voydeya are currently under review with multiple global health authorities.
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