Voydeya approved in Canada as add-on therapy for PNH group
Taken with Soliris or Ultomiris, it controls anemia due to extravascular hemolysis
Health Canada has approved the oral therapy Voydeya (danicopan) as an add-on to Ultomiris (ravulizumab) or Soliris (eculizumab) in adults with paroxysmal nocturnal hemoglobinuria (PNH) who have residual anemia due to blood cell destruction taking place outside blood vessels.
The announcement came just a few months after Voydeya received similar approvals in the U.S., European Union, and Japan.
“People living with a rare disease and their families deserve our unwavering commitment to developing and enabling access to therapies that can help them live longer, fuller lives,” Karen Heim, general manager of Alexion Canada, said in a company press release. Voydeya, Ultomiris, and Soliris are marketed by Alexion, AstraZeneca Rare Disease.
Add-on PNH treatment allowing for dual complement inhibition
PNH is caused by blood cells lacking certain surface proteins that work as an identification badge, signaling to the immune system that these are healthy cells. As a result, a part of the immune system called the complement cascade becomes abnormally activated, leading to blood cell destruction.
Ultomiris and Soliris are standard-of-care treatments that work to block complement activation by targeting a protein called C5. These therapies are generally quite effective for preventing red blood cell destruction occurring within blood vessels (intravascular hemolysis). Yet, some patients have persistent PNH symptoms due to red blood cell destruction taking place outside blood vessels (extravascular hemolysis).
Voydeya helps to control extravascular hemolysis by blocking the activity of another complement protein called factor D.
Voydeya’s latest approval “is very exciting news for Canadian PNH patients,” said Barry Katsof, founder and president of the Canadian Association of PNH Patients. Its approval “offers the subset of patients experiencing extravascular hemolysis while treated with [Soliris] or [Ultomiris] an add-on therapy designed to address this condition and improve their quality of life.”
Christopher Patriquin, chair of the Canadian PNH Network, added that “dual complement inhibition can enable patients to remain on our current standard of care C5 inhibition to maintain disease control, while increasing hemoglobin and potentially further improving their quality of life.” Hemoglobin is the protein that carries oxygen through the bloodstream.
Voydeya’s approvals were based mainly on data from a Phase 3 clinical trial called ALPHA (NCT04469465), which tested the add-on therapy against a placebo in 86 patients experiencing substantial extravascular hemolysis despite treatment with Soliris or Ultomiris.
Results showed the oral therapy worked to ease anemia and lessen the need for blood transfusions, with consistent results seen after up to a year of treatment.
The most common side effect of Voydeya is headache. Since the therapy works by suppressing a part of the immune system, Voydeya can increase the risk of serious and potentially life-threatening infections, particularly those caused by encapsulated bacteria.
About the Author
