RARE Daily

EU Approves AstraZeneca’s Voydeya for PNH as Add-on

April 23, 2024

Rare Daily Staff

The European Union approved AstraZeneca’s Voydeya as an add-on to Ultomiris and Soliris for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria, a rare, chronic, progressive and potentially life-threatening blood disorder.

Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by red blood cell destruction within blood vessels and white blood cell and platelet activation, which can result in thrombosis (blood clots). It is caused by an acquired genetic mutation that may happen any time after birth and results in the production of abnormal blood cells that are missing important protective blood cell surface proteins. These missing proteins enable the complement system, which is part of the immune system and is essential to the body’s defense against infection, to attack and destroy or activate these abnormal blood cells.

Living with PNH can be debilitating, and signs and symptoms may include blood clots, abdominal pain, difficulty swallowing, erectile dysfunction, shortness of breath, excessive fatigue, anemia and dark-colored urine.

Voydeya is a first-in-class, oral, factor D inhibitor. The medication works by selectively inhibiting Factor D, a complement system protein that plays a key role in the amplification of the complement system response. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. Voydeya has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration and PRIority MEdicines (PRIME) status by the European Medicines Agency. Voydeya has also been granted Orphan Drug designation in the United States, European Union, and Japan for the treatment of PNH.

Voydeya as an add-on to standard-of-care Ultomiris (ravulizumab) or Soliris (eculizumab) is designed to address the needs of the approximately 10 to 20 percent of patients with PNH who experience clinically significant extravascular hemolysis (EVH) while treated with a C5 inhibitor.

“The EC approval of Voydeya as an add-on therapy represents an important innovation for the subset of patients with PNH who experience continued symptoms of anemia due to EVH while treated with a C5 inhibitor,” Hubert Schrezenmeier, medical director at the Institute of Transfusion Medicine at The University of Ulm, said. “These patients now have an option to address the manifestations of EVH while remaining on treatment with standard-of-care C5 inhibitor therapy, Soliris or Ultomiris, to maintain disease control and help prevent the potentially life-threatening complications that can be associated with this devastating disease.”

Photo: Hubert Schrezenmeier, medical director at the Institute of Transfusion Medicine at The University of Ulm

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