AstraZeneca gets FDA nod for rare blood disease therapy

April 1, 2024

The U.S. FDA has approved AstraZeneca's oral Factor D inhibitor as an add-on therapy to ravulizumab or eculizumab for a subset of adults with the rare blood disease, paroxysmal nocturnal hemoglobinuria.

Voydeya (danicopan) is approved as an add-on to the standard-of-care — AstraZeneca's C5 inhibitors, Ultomiris (ravulizumab) or Soliris (eculizumab) — to address the needs of the approximately 10-20% of patients with PNH who experience clinically significant extravascular hemolysis (EVH) while treated with a C5 inhibitor. For some patients, EVH, which is the destruction of red blood cells outside of the blood vessels, results in continued symptoms of anemia and may require blood transfusions.

AstraZeneca picked up danicopan in 2021 through its $39 billion mega buyout of Alexion after Alexion acquired the drug through its $930 million buyout of Achillion Pharmaceuticals in 2019.

Voydeya will go up against Novartis' Factor B inhibitor, branded Fabhalta, which was approved as the first oral monotherapy for the treatment of adults with PNH back in December. According to Novartis, Fabhalta addresses the unmet needs of patients being treated with AstraZeneca's Ultomiris or Soliris,  including EVH. Fabhalta was tested against Ultomiris or Soliris in the open-label APPLY-PNH trial, where the drug proved superiority.

But, AstraZeneca's trial results for Voydeya suggest that a dual complement pathway inhibition at Factor D and C5 may be an optimal treatment approach for EVH patients.

The approval for AstraZeneca comes just days after the FDA approved a label expansion for Ultomiris, for the treatment of neuromyelitis optica spectrum disorder (NMOSD). First approved in 2018 as a treatment for PHN, Ultomiris has notched subsequent approvals in hemolytic uremic syndrome (a rare condition that leads to progressive kidney failure), generalized myasthenia gravis (a chronic autoimmune disorder), and now, neuromyelitis optica spectrum disorder.