AstraZeneca Voydeya approved in Japan for paroxysmal nocturnal haemoglobinuria

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.

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. 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.”

Marc Dunoyer, Chief Executive Officer, Alexion, said, “More than 20 years of PNH research has cemented the role of C5 inhibition in effectively treating this rare disease, and we continue to innovate for this community. Voydeya, as add-on to standard-of-care, is a testament to our determination to address the needs of those impacted by clinically significant EVH without disruption to proven therapy. We look forward to bringing this important advancement to the subset of PNH patients in Japan who are living with this condition.”

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 haemoglobin ≤9.5 g/dL and absolute reticulocyte count levels ≥120x109/L. Results showed that Voydeya met the primary endpoint of change in haemoglobin 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. 

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.