Voydeya (danicopan) has been granted approval in the United States as an add-on therapy for the treatment of extravascular haemolysis (EVH) in adults diagnosed with paroxysmal nocturnal haemoglobinuria (PNH). This milestone marks a significant advancement in addressing the needs of individuals grappling with this rare and potentially life-threatening blood disorder.

The approval by the US Food and Drug Administration (FDA) follows rigorous evaluation, predominantly based on the remarkable outcomes derived from the pivotal ALPHA Phase III trial. Published results from this trial, which spanned a 12-week primary evaluation period and were featured in The Lancet Haematology, underscored the efficacy and safety of Voydeya as a complement to standard-of-care therapies such as Ultomiris (ravulizumab) or Soliris (eculizumab).

Dr. Bart Scott, a distinguished figure in hematology and oncology, expressed his enthusiasm, noting, "The approval of Voydeya offers this small subset of PNH patients an add-on therapy designed to address EVH, while maintaining disease control with Ultomiris or Soliris."

Voydeya, a first-in-class oral Factor D inhibitor, has garnered considerable attention owing to its potential to revolutionize PNH treatment paradigms. Marc Dunoyer, CEO of Alexion, remarked, "The approval of first-in-class, Factor D inhibitor Voydeya marks an important advancement in the treatment of PNH and builds on our leadership and commitment to bring forward innovation in complement science."

The ALPHA Phase III trial not only met its primary endpoint of change in haemoglobin from baseline to week 12 but also demonstrated promising results across key secondary endpoints, including transfusion avoidance and improvements in Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-Fatigue) scores.

Furthermore, the safety profile of Voydeya was deemed favorable, with no notable concerns identified during the trial. Common treatment-emergent adverse events included headache, nausea, arthralgia, and diarrhoea.

With Breakthrough Therapy designation from the FDA, PRIority MEdicines (PRIME) status from the European Medicines Agency, and Orphan Drug Designation across multiple regions, Voydeya's approval represents a significant stride forward in addressing the unmet needs of PNH patients worldwide.

The approval of Voydeya not only exemplifies the potential of innovative therapies but also underscores the commitment of stakeholders in advancing care for individuals grappling with rare diseases.

As regulatory reviews continue in various regions, the approval of Voydeya stands as a beacon of hope for those affected by PNH, reaffirming the relentless pursuit of transformative treatments in the field of rare diseases.