FDA Approves Voydeya as Add-on Therapy in Rare Blood Disorder

The FDA has approved Alexion/AstraZeneca’s Voydeya (danicopan) as add-on therapy to Ultomiris (ravulizumab) or Soliris (eculizumab) to treat adult patients with paroxysmal nocturnal hemoglobinuria (PNH). It is indicated for those patients with extravascular hemolysis (EVH) where red blood cells are destroyed outside of the blood vessel despite treatment with Soliris or Ultomiris.

PNH is a rare, chronic and progressive blood disorder. It is characterized by red blood cell destruction within blood vessels and white blood cell and platelet activation, which can result in thrombosis. It is caused by an acquired genetic mutation that can occur any time after birth. This mutation results in the production of abnormal blood cells that are missing important proteins.

The current standard of care for paroxysmal nocturnal hemoglobinuria (PNH) are the C5 inhibitors Soliris and Ultomiris, which are monoclonal antibodies designed to target the complement protein C5. The complement system plays a role in the immune system, but inappropriate activation can lead to disorders such as PNH. Both Soliris and Ultomiris were developed by Alexion, part of AstraZeneca.

Voydeya is a first-in-class, oral, Factor D inhibitor developed as an add-on to standard-of-care Solis or Ultomiris to address the needs of about 10% to 20% of patients with PNH who experience clinically significant extravascular hemolysis while treated with a C5 inhibitor. The companies have not indicated when Voydeya will be available or what the list price is.

For the full year 2023, Soliris generated worldwide sales of $3.1 billion; Ultomiris generated worldwide sales of $2.9 billion. The list price of Soliris is $6,878.22 for 3 mL, according to Drugs.com. The price of Ultomiris is $6,752.91 for 3 mL, according to Drugs.com.

Bart Scott, M.D.

“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,” Bart Scott, M.D., professor, division of Hematology and Oncology at the University of Washington Medical Center, and professor, Clinical Research Division at Fred Hutchinson Cancer Center, said in a news release.

The approval of Voydeya was based on positive results from the pivotal ALPHA phase 3 trial, which showed a clinically meaningful increase in hemoglobin levels and maintained disease control in patients with PNH.

Results from the 12-week primary evaluation period of the trial were published in December 2023 in The Lancet Haematology.

Separately, the companies announced that Ultomiris has also been approved to treat adults with neuromyelitis optica spectrum disorder, an autoimmune disorder that primarily impacts the optic nerves, brain stem and spinal cord. Patients with this disorder experience relapses and the onset of new symptoms or worsening symptoms.

In the phase 3 CHAMPION-NMOSD trial, patients with treated with Ultomiris saw a 98.6% reduction in risk of relapse at 73 weeks. These results were published in the March 2023 Annals of Neurology.