A new option for adult patients with paroxysmal nocturnal hemoglobinuria (PNH) has been introduced in Korea with the approval of Voydeya (danicopan), a first-in-class oral factor D inhibitor.

Voydeya is AstraZeneca Korea's PNH treatment that was approved by the Ministry of Food and Drug Safety (MFDS) on March 28th.

Voydeya is a first-in-class oral factor D inhibitor and can be used as an add-on therapy to ravulizumab (Ultomiris) or eculizumab (Soliris) for PNH patients with symptoms or signs of extravascular haemolysis (EVH) i in patients who are already receiving a C5 inhibitor (ravulizumab or eculizumab).

PNH is a rare disease caused by acquired genetic mutation that results in hemolysis and thrombosis, which can cause anemia, fatigue, hemoglobinuria, and even death.

PNH Is treated with C5 inhibitors ravulizumab or eculizumab, which can reduce intravascular hemolysis and thrombosis. However, extravascular hemolysis can occur if defective red blood cells accumulate on C3 during treatment.

The MFDS approval was based on the phase III ALPHA clinical trial. The study confirmed the superiority of Voydeya as add-on therapy in adult PNH patients treated with ravulizumab or eculizumab who developed significant EVH (haemoglobin ≤9.5 g/dL; absolute reticulocyte count ≥120 × 10⁹/L).

Results showed that Voydeya met both the primary and secondary endpoints. The primary endpoint, change in hemoglobin concentration from baseline to week 12, was 2.94 g/dL for Voydeya versus 0-50 g/dL for placebo.

The least squares mean (LSM) difference between Voydeya and placebo was 2.44 g/dL (95% CI 1.69 to 3.20; p<0.0001). In addition, the difference in mean hemoglobin concentration between Voydeya and placebo was seen since Week 1 of treatment.

In addition, 60% (25 of 42) of patients achieved a transfusion-free hemoglobin increase of 2 g/dL or more at Week 12, compared to 0 with placebo.

Chul Woong Kim, Director of the Rare Disease Business Unit at AstraZeneca Korea, said, “The ALPHA trial has been made possible with the contributions of Korean medical experts, including Dr. Jong-Wook Lee, Professor of Hematology/Oncology at Hanyang University Hospital, as first author. We are pleased to be able to address the unmet need for PNH patients whose quality of life is compromised by extravascular hemolysis."