Voydeya (danicopan), a first-in-class oral Factor D inhibitor from Alexion, AstraZeneca Rare Disease, has received approval from the European Commission as an add-on treatment to standard-of-care therapies ravulizumab (Ultomiris) or eculizumab (Soliris) for adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who suffer from residual haemolytic anaemia. This approval marks a significant advancement for the approximately 10-20% of PNH patients who continue to experience clinically significant extravascular haemolysis (EVH), despite treatment with a C5 inhibitor.

The approval was based on the positive outcomes from the pivotal ALPHA Phase III trial, the results of which were published in The Lancet Haematology. During the 12-week primary evaluation period, Voydeya demonstrated significant efficacy in managing symptoms and improving patient outcomes. It met its primary endpoint of change in haemoglobin from baseline to week 12 and achieved all key secondary endpoints, including transfusion avoidance and improvement in the Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-Fatigue) score.

Professor Hubert Schrezenmeier, MD, Medical Director at the Institute of Transfusion Medicine at The University of Ulm, emphasized the importance of this approval, stating: “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.” He highlighted the potential of Voydeya to maintain disease control and help prevent life-threatening complications associated with PNH.

Marc Dunoyer, Chief Executive Officer of Alexion, expressed enthusiasm about the new treatment option, noting, “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.” He confirmed Alexion’s commitment to making Voydeya accessible to patients in Europe and furthering global access.

Voydeya has already been approved in the US and Japan and is under review in other countries. It has received Breakthrough Therapy designation by the US Food and Drug Administration and PRIority MEdicines (PRIME) status by the European Medicines Agency. Additionally, it holds Orphan Drug Designation in the US, EU, and Japan for the treatment of PNH. Alexion is also exploring the potential of Voydeya as a monotherapy for geographic atrophy in a Phase II clinical trial.

The approval of Voydeya in the European Union as an add-on therapy for PNH represents a significant step forward in the management of this rare disease. By addressing the unmet needs of patients suffering from EVH, Voydeya enhances the existing treatment landscape and offers hope for improved patient outcomes. This development not only highlights the continuous innovation in the treatment of rare diseases but also emphasizes the importance of targeted therapies in achieving better health standards.

  Alexion Pharmaceuticals, AstraZeneca, clinical trials, European Commission, PNH treatment, residual haemolytic anaemia, Voydeya approval