AstraZeneca's First-in-Class Oral Factor D Inhibitor Voydeya (Danicopan) Receives World’s First Approval in Japan for PNH Patients with Residual Extravascular Hemolysis

On January 19, AstraZeneca announced that its first-in-class oral complement factor D inhibitor, Voydeya (danicopan), had been approved for marketing in Japan, achieving the world's first approval. It is used to treat patients with paroxysmal nocturnal hemoglobinuria (PNH). When patients have an insufficient response to C5 inhibitors, it can be used in combination with C5 inhibitor therapy to address the needs of PNH patients (approximately 10-20%) who experience clinically significant extravascular hemolysis (EVH) while receiving C5 inhibitor treatment.

Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare and serious blood disorder characterized by the destruction of red blood cells within blood vessels (also known as intravascular hemolysis) and the activation of white blood cells and platelets, which can lead to thrombosis. In patients with PNH, the complement system is abnormally activated; therefore, treatment can be achieved through C5 complement inhibitors. However, patients receiving C5 complement inhibitor therapy often experience clinically significant extravascular hemolysis (EVH).

Danicopan is an oral medication used as an add-on to C5 inhibitor therapies Ultomiris or Soliris. The drug selectively inhibits complement factor D, which plays a key role in the amplification of the complement system response.

The approval by Japan's Ministry of Health, Labour and Welfare (MHLW) was based on positive results from the pivotal ALPHA Phase III trial. The results of the 12-week initial evaluation period of the trial have been published in The Lancet Haematology.

ALPHA is a randomized, placebo-controlled Phase III study that enrolled a total of 84 patients with paroxysmal nocturnal hemoglobinuria who experienced clinically significant extravascular hemolysis. The results showed that, compared with the placebo group, patients in the Danicopan group had a statistically significant and clinically meaningful improvement in hemoglobin levels from baseline at week 12, achieving the primary endpoint of the study. The study also met key secondary endpoints, including avoidance of transfusions and changes in FACIT fatigue scores. Overall, Danicopan demonstrated good safety and tolerability.

It is worth mentioning that this drug was originally developed by Alexion. In 2020, AstraZeneca acquired the company for approximately $39 billion, gaining access to a variety of complement drugs and innovative therapies for rare diseases.

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