Welireg FDA Approval History
Last updated by Judith Stewart, BPharm on May 15, 2025.
FDA Approved: Yes (First approved August 13, 2021)
Brand name: Welireg
Generic name: belzutifan
Dosage form: Tablets
Company: Merck
Treatment for: Von Hippel-Lindau Syndrome, Renal Cell Carcinoma, Pheochromocytoma
Welireg (belzutifan) is a selective inhibitor of hypoxia-inducible factor 2 alpha (HIF-2α) for the treatment of von Hippel-Lindau (VHL) disease, advanced renal cell carcinoma, and pheochromocytoma or paraganglioma (PPGL).
- Welireg is indicated:
von Hippel-Lindau (VHL) disease
- for treatment of adult patients 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), not requiring immediate surgery.
Advanced Renal Cell Carcinoma (RCC)
- for treatment of adult patients with advanced renal cell carcinoma (RCC) with a clear cell component following a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and a vascular endothelial growth factor tyrosine kinase inhibitor (VEGF-TKI).
Pheochromocytoma or Paraganglioma (PPGL)
- for treatment of adult and pediatric patients 12 years and older with locally advanced, unresectable, or metastatic pheochromocytoma or paraganglioma (PPGL). - - Von Hippel-Lindau disease is a rare genetic disease caused by a genetic mutation in the VHL tumor suppressor gene. When VHL is inactivated, proteins known as hypoxia-inducible factors can accumulate, leading to the growth of both benign and malignant tumors.
- Advanced renal cell carcinoma is renal cell carcinoma that has spread to another part of the body.
- Pheochromocytoma and paraganglioma are rare tumors that can be caused by certain genetic syndromes or mutations. Pheochromocytoma form in nerve tissue in the center of the adrenal gland, whereas paraganglioma form in nerve tissue near certain blood vessels and nerves outside the adrenal glands. - Welireg works by reducing transcription and expression of hypoxia-inducible factor 2 alpha (HIF-2α) target genes associated with cellular proliferation, angiogenesis and tumor growth.
- Welireg tablets are administered once daily with or without food.
- Welireg may cause serious side effects including anemia and hypoxia.
- Common (≥25%) adverse reactions:
- in VHL disease, including laboratory abnormalities, include decreased hemoglobin, fatigue, increased creatinine, headache, dizziness, increased glucose, and nausea.
- in advanced RCC, including laboratory abnormalities, include decreased hemoglobin, fatigue, musculoskeletal pain, increased creatinine, decreased lymphocytes, increased alanine aminotransferase, decreased sodium, increased potassium, and increased aspartate aminotransferase.
- in PPGL, including laboratory abnormalities, include anemia, fatigue, musculoskeletal pain, decreased lymphocytes, increased alanine aminotransferase, increased aspartate aminotransferase, increased calcium, dyspnea, increased potassium, decreased leukocytes, headache, increased alkaline phosphatase, dizziness, and nausea.
Development timeline for Welireg
Further information
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