Voydeya (danicopan) has been approved in Japan for the treatment of paroxysmal nocturnal haemoglobinuria (PNH).- AstraZeneca

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 haemolysis (EVH) while treated with a C5 inhibitor.

The approval by the Japanese Ministry of Health, Labour 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 ( previously cited).

PNH is a rare and severe blood disorder characterised by the destruction of red blood cells within blood vessels, known as intravascular haemolysis (IVH), and white blood cell and platelet activation that can cause thrombosis (blood clots) and result in organ damage and potentially premature death. 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 anaemia and require blood transfusions.

Professor Jun-ichi Nishimura, MD, PhD, Department of Haematology and Oncology at Osaka University Graduate School of Medicine, said: “C5 inhibition has transformed care for PNH and is proven to control IVH, prevent complications and improve survival. A small subset of patients treated with C5 inhibitor therapy may experience signs or symptoms of clinically significant EVH. In the ALPHA trial, Voydeya as an add-on to Ultomiris or Soliris improved haemoglobin levels and reduced the need for transfusions, while maintaining control of IVH. Today’s approval has the potential to improve outcomes for those experiencing the burdensome manifestations of EVH while continuing treatment with standard of care".