On May 14, 2025, Merck announced that the U.S. Food and Drug Administration (FDA) has approved Welireg (belzutifan) for the treatment of adult and pediatric patients aged 12 years and older with locally advanced, unresectable, or metastatic pheochromocytoma or paraganglioma (PPGL).
The press release states that Welireg is the first oral treatment approved by the FDA for pheochromocytoma and paraganglioma.
Welireg Indications and Uses
Welireg is a hypoxia-inducible factor inhibitor indicated for:
1. Lindau Syndrome
• For the treatment of adult patients with Lindau Syndrome who do not require immediate surgery and require treatment for related renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastoma, or pancreatic neuroendocrine tumor (pNET).
2. Advanced Renal Cell Carcinoma
• For use in adult patients with advanced renal cell carcinoma treated with programmed death receptor 1 (PD-1) or programmed death ligand 1 (PD-L1) inhibitors and vascular endothelial growth factor tyrosine kinase inhibitors (VEGF-TKIs).
