AstraZeneca, which bought the drug’s developer, Alexion Pharmaceuticals, in 2021, won approval from the U.S. Food and Drug Administration (FDA) on March 29, 2024, for Voydeya^™ (danicopan) tablets. It is the first drug in its therapy class, complement factor D inhibitors, to be approved in the U.S. Together with one of AstraZeneca’s complement 5 (C5) inhibitors — eitherUltomiris^® (ravulizumab-cwvz) injection or Soliris^® (eculizumab) injection – it is indicated to treat adults who experience extravascular hemolysis (EVH) associated with paroxysmal nocturnal hemoglobinuria (PNH). The recommended dose is 150mg three times a day, with an increase to 200mg three times a day, if needed. A boxed warning cautions that specific types of bacteria may cause severe infections for patients who take Voydeya. Due to the risk, it will be dispensed only under a risk evaluation and mitigation strategy (REMS). A launch date and pricing plans have not yet been announced. Look here for its prescribing information. 

At a Glance

• Brand (Generic) Name: Voydeya(danicopan)
• Manufacturer: AstraZeneca
• Date Approved: March 29, 2024
• Indication: as add-on therapy to ravulizumab or eculizumab for the treatment of EVH for adults who have PNH
• Dosage Forms Available: 50mg and 100mg oral tablets
• Launch Date: Not yet determined
• Estimated Cost: Not available
• PNH is a blood disorder caused by spontaneous mutations of a protein that attracts and holds other proteins, including some that protect against the immune responses, onto the surfaces of red blood cells (RBCs).
• The RBCs of patients who have PNH are easily destroyed by immune proteins called complement — both within blood vessels (intravascular/IVH) and in the liver, lymph nodes, and spleen (EVH).
• Hemoglobin (Hgb) levels, which normally range from 14 to 18gm/dL for men and 12 to 16gm/dL for women, typically are decreased by PNH. 
• Up to one-half of patients who receive the current standard of therapy for PNH with a C3 or C5 inhibitor still need blood transfusions and a large percentage have persistent anemia because currently available complement inhibitors are not very effective for EVH.
• PNH is believed to affect between 0.5 and 1.5 people per one million in the general population — fewer than 500 patients in the United States. AstraZeneca estimates that up to 20% of patients still have EVH despite being treated with a C5 inhibitor.
• By interrupting Factor D, part of the alternative complement cascade, Voydeya acts differently from other complement inhibitors to manage PNH.
• In its phase III clinical trial, patients taking Voydeya averaged a 2.9gm/dL increase in Hgb, and 83.3% did not need transfusions after 12 weeks of treatment as compared to a 0.5gm/dL increase and 38.1% for those taking placebo tablets. All patients also received infusions of either Ultomiris or Soliris.
• Because taking Voydeya may increase the risk of serious infections with some kinds of bacteria, patients using it should have any vaccinations for bacterial illnesses at least two weeks before starting treatment.
• Fabhalta^® (iptacopan) tablets, the first factor B complement inhibitor, was FDA-approved in December 2023, as monotherapy for both IVH and EVH.
• In May 2021, a C3 inhibitor, Empaveli^® (pegcetacoplan) was approved by the FDA. It is infused subcutaneously (SC) twice a week to manage both IVH and EVH associated with PNH.
• Voydeya was approved as a Breakthrough Therapy and was granted Orphan Drug status.