AstraZeneca Korea’s oral D-factor inhibitor Voydeya approved for PNH treatment

The Ministry of Food and Drug Safety (MFDS) approved AstraZeneca Korea's oral D-factor inhibitor, Voydeya (ingredient: danicopan), as an adjunct therapy for treating extravascular hemolysis in adult patients with paroxysmal nocturnal hemoglobinuria (PNH).

PNH is a rare, genetically driven disorder characterized by hemolysis and thrombosis, leading to anemia, fatigue, hemoglobinuria, and potentially death.

Current treatments focus on reducing intravascular hemolysis and thrombosis using C5 inhibitors such as ravulizumab or eculizumab. However, these treatments can result in extravascular hemolysis if defective red blood cells accumulate in C3.

This severe condition necessitates blood transfusions and occurs in approximately 10-20 percent of PNH patients.

Voydeya, the first oral D-factor inhibitor, received MFDS approval based on its efficacy in addressing extravascular hemolysis symptoms in PNH patients already receiving C5 inhibitors (ravulizumab or eculizumab).

The approval was supported by the ALPHA study, a randomized, double-blind, multi-center phase 3 clinical trial involving adult PNH patients experiencing significant extravascular hemolysis.

The ALPHA study demonstrated Voydeya’s superiority in meeting both primary and secondary endpoints.

At week 12, Voydeya showed a mean increase in hemoglobin levels of 2.94 g/dL compared to a placebo increase of 0.50 g/dL, with a least squares mean difference of 2.44 g/dL.

Notably, the trial data showed that the hemoglobin level difference between Voydeya and placebo was evident from the first week of treatment.

Additionally, 60 percent of patients (25 out of 42) treated with Voydeya achieved a hemoglobin increase of at least 2 g/dL without transfusion by week 12, compared to none in the placebo group.

Also, 83 percent of Voydeya-treated patients avoided transfusions during the 12-week period, significantly higher than the 38 percent in the placebo group.

Voydeya also improved patient-reported fatigue scores (FACIT-Fatigue) by an average of 7.97 points compared to a 1.85-point improvement in the placebo group. The most common adverse reaction was headache, with no serious drug-related adverse events reported.

"The ALPHA study, in which Korean physicians, including Professor Jong-Wook Lee from the Hematology and Oncology Department at Hanyang University Hospital, participated as the lead author, is a treatment born out of significant contributions from local medical staff," AstraZeneca Korea’s Rare Disease Business Unit Director Kim Chul-woong said. "We are pleased to be able to meet the unmet needs of PNH patients whose quality of life has been lowered by extravascular hemolysis, and we will continue to strive to ensure that domestic rare disease patients can receive treatment in a better environment."