London – Voydeya (danicopan) has been approved in the European Union (EU) as an add-on to ravulizumab or eculizumab for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have residual haemolytic anaemia.1 Voydeya is a first-in-class, oral, Factor D inhibitor developed as an add-on to standard-of-care Ultomiris (ravulizumab) or Soliris (eculizumab) to address the needs of the approximately 10-20% of patients with PNH who experience clinically significant extravascular haemolysis (EVH) while treated with a C5 inhibitor.2,3
The approval by the European Commission (EC) follows the positive opinion of the Committee for Medicinal Products for Human Use and is based on 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.2
Professor Hubert Schrezenmeier, MD, Medical Director, Institute of Transfusion Medicine at The University of Ulm, said: “The EC approval of Voydeya as an add-on therapy represents an important innovation for the subset of patients with PNH who experience continued symptoms of anaemia due to EVH while treated with a C5 inhibitor. These patients now have an option to address the manifestations of EVH while remaining on treatment with standard-of-care C5 inhibitor therapy, Soliris or Ultomiris, to maintain disease control and help prevent the potentially life-threatening complications that can be associated with this devastating disease.”
Marc Dunoyer, Chief Executive Officer, Alexion, said: “For patients with PNH, Voydeya as an add-on therapy has been shown to address signs and symptoms of clinically significant EVH, including anaemia, while maintaining standard-of-care treatment with Soliris or Ultomiris. We look forward to making this first-in-class Factor D inhibitor available to patients in Europe and to advancing access around the globe.”
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 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.2
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.2
Voydeya has been granted Breakthrough Therapy designation by the United States (US) Food and Drug Administration (FDA) and PRIority MEdicines (PRIME) status by the European Medicines Agency (EMA). Voydeya has also been granted Orphan Drug Designation in the US, EU and Japan for the treatment of PNH. Voydeya has been approved in the US and Japan, and regulatory reviews are ongoing in additional countries.