Don Tracy, Associate Editor

Treatment approved in combination with ravulizumab or eculizumab, representing minority demographic still suffering after receiving C5 inhibitor therapy for extravascular haemolysis.
Alexion, AstraZeneca Rare Disease announced that the FDA has approved Voydeya (danicopan) as an add-on therapy for the treatment of extravascular haemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH), in combination with ravulizumab or eculizumab. According to the company, the medication treats patients with significant EVH despite receiving C5 inhibitor therapy. The approval was based on positive results from the Phase III ALPHA trial, which found that Voydeya improved hemoglobin levels while providing the benefit of avoiding transfusions after a primary 12-week period. Additionally, there was no significant increases reported related to safety concerns, with common adverse reactions including headache, nausea, arthralgia, and diarrhea.1
“The approval of first-in-class, Factor D inhibitor Voydeya marks an important advancement in the treatment of PNH and builds on our leadership and commitment to bring forward innovation in complement science,” said Marc Dunoyer, CEO, Alexion, AstraZeneca Rare Disease, in a press release. “As the ALPHA trial suggests, dual complement pathway inhibition at Factor D and C5 may be an optimal treatment approach for this subset of patients with EVH, enabling them to continue with proven standard-of-care therapy.”
Back in February, the Committee for Medicinal Products for Human Use of the European Medicines Agency gave a positive recommendation for use of Voydeya in the EU, also based on results stemming from the ALPHA study, while the Japanese Ministry of Health, Labour and Welfare approved Voydeya for the treatment of PNH in combination with C5 inhibitor therapy when patients have had an insufficient response to these medications.2,3
“The approval of Voydeya offers this small subset of PNH patients an add-on therapy designed to address EVH, while maintaining disease control with Ultomiris or Soliris. Terminal complement inhibition with Ultomiris can address the life-threatening complications of PNH, building on the efficacy and safety of Soliris established over nearly 20 years,” said Bart Scott, MD, professor, division of hematology and oncology, University of Washington Medical Center, professor, clinical research division, Fred Hutchinson Cancer Center, in the press release.
Hemolysis can be caused my multiple factors, which include the following:
Defective red blood cells
Issues with the immune system
Infections
Use of certain medications
Complications with pregnancy
Toxins
Use of medical devices4
“Extravascular hemolysis involves red blood cell destruction that takes place outside of your blood vessels,” the Cleveland Clinic noted.” Blood vessels include veins, arteries and capillaries. Your spleen is the most common site of extravascular hemolysis. As blood filters through your spleen, white blood cells called macrophages locate old or damaged red blood cells and destroy them.”