Belzutifan, sold under the brand name Welireg, is an anti-cancer medication used for the treatment of von Hippel–Lindau disease-associated renal cell carcinoma.
It is taken by mouth.
Belzutifan is an hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor.
It has the capacity to reduce serum erythropoietin verified its clinical applicability for the treatment of malignancies linked to von Hippel-Lindau (VHL), such as renal cell carcinoma (RCC) with clear cell histology (ccRCC), pancreatic lesions, neuroendocrine tumors, and CNS hemangioblastomas or pancreatic neuroendocrine tumors (pNET).
Belzutifan obtained a disease control rate of 80% in pretreated ccRCC during a phase I trial.
The most common side effects include decreased hemoglobin, anemia, fatigue, increased creatinine, headache, dizziness, increased glucose, and nausea.
Belzutifan is the first hypoxia-inducible factor-2 alpha inhibitor therapy approved in the US.
The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.
Belzutifan is indicated for treatment of adults with von Hippel-Lindau (VHL) disease who require therapy for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET).
Belzutifan is well tolerated: Anemia, tiredness, headaches, vertigo, nausea, and dyspnea were the most typical side effects.
All participants in the phase II trial reported a hemoglobin reduction of at least 1.9 g/dL; however, only a small number of individuals needed transfusions or erythropoietin-stimulating medications.
Anemia was a predicted negative outcome of inhibiting the EPO gene.
Belzutifan has the potential to harm fetuses and embryos during pregnancy and can render some hormonal contraceptives ineffective.