AstraZeneca adds to rare blood disease portfolio with world-first Voydeya approval in Japan

A month after Novartis made its entry into the paroxysmal nocturnal haemoglobinuria (PNH) field, AstraZeneca’s newest contender Voydeya bolsters the company’s presence in the disease area with a world-first approval in Japan.

Voydeya (danicopan), a first-in-class oral factor D inhibitor, won backing from the Japanese Ministry of Health, Labour and Welfare (MHLW) for use alongside a C5 inhibitor to treat PNH patients who have had an insufficient response to C5 inhibitors. It’s a new option for the 10% to 20% of patients with PNH who have experienced clinically significant extravascular haemolysis (EVH) after C5 inhibitor treatment, AZ notes.

Voydeya was designed as a potential add-on to AstraZeneca's established C5 inhibitor therapies Ultomoris or Soloris, which represent the current standard of care. In its ALPHA phase 3 trial, Voydeya was able to change hemoglobin levels from baseline to week 12 and meet secondary endpoints, including transfusion avoidance.

Long-term follow-up data from the study, which the company presented at December, showed that the improvements in mean hemoglobin levels and absolute reticulocyte count levels held up through 48 weeks. Plus, 83% of patients didn’t need a blood transfusion after 12 weeks, while 78% of patients could say the same after 24 weeks.

“Voydeya, as add-on to standard-of-care, is a testament to our determination to address the needs of those impacted by clinically significant EVH without disruption to proven therapy,” Marc Dunoyer, CEO of AstraZeneca’s rare disease subsidiary Alexion, said in a press release. 

Alexion has long dominated the PNH market with Ultomiris and Solaris. Competitors, such as Apellis’ Empaveli, have entered the ring recently, while Novartis’ factor B inhibitor Fabhalta (iptacopan) scored its own FDA approval in December.

Fabhalta marked the first oral monotherapy approved in the U.S. for the rare blood disorder. Both Soloris and Ultomoris are injections.

Voydeya, meanwhile, carries an FDA breakthrough therapy designation in the U.S. It also received priority medicines status from the European Medicines Agency and is under review with “multiple global health authorities,” AstraZeneca noted in its release.  

The company is also studying the med as a potential monotherapy for geographic atrophy in a phase 2 trial.