AstraZeneca (AZ; NASDAQ: AZN), a UK-based pharmaceutical giant, has reportedly secured market approval in South Korea for its drug Voydeya (danicopan), the world’s first-in-class oral D-factor inhibitor. The Ministry of Food and Drug Safety (MFDS) in Korea approved Voydeya for use as an adjunct therapy for extravascular hemolysis (EVH) in adults with paroxysmal Nocturnal Hemoglobinuria (PNH), to be used alongside treatment with C5 inhibitors ravulizumab or eculizumab.
Voydeya, discovered by Achillion Pharmaceuticals and later acquired by Alexion Pharmaceutical in 2019, became part of AstraZeneca’s portfolio following a $39 billion takeover completed in 2021. This approval in Korea comes on the heels of Voydeya’s approval by the US FDA in April 2024 for the same indication, serving as an add-on treatment to ravulizumab (AZ’s Ultromiris) or eculizumab (AZ’s Soliris) for treating EVH in PNH patients. PNH is a rare, genetically driven disorder characterized by anemia, fatigue, and potentially fatal complications.
The approval of Voydeya was supported by the positive results of the Phase III ALPHA study, which involved adult PNH patients and met both its primary and secondary endpoints. Voydeya demonstrated a mean increase in hemoglobin levels of 2.94 g/dL compared to a placebo increase of 0.50 g/dL. By week 12, 60% of patients (25 out of 42) treated with Voydeya achieved a hemoglobin increase of at least 2 g/dL without transfusion, compared to none in the placebo group. Moreover, 83% of patients treated with Voydeya avoided transfusions during the 12-week period, a significant improvement over the 38% rate in the placebo group. Danicopan is currently undergoing Phase III trials in China.- Flcube.com
