Analysis of the clinical application and effect of Bestifan (Viliru) on other rare tumors

Belzutifan (Belzutifan, trade nameWelireg) is a targeted inhibitor of HIF-2α Oral drug, initially used to treat tumors such as clear cell renal cell carcinoma (ccRCC) associated with von Hippel syndrome (VHL). In recent years, Bestifan has made significant progress in clinical application and efficacy research in a variety of rare tumors, especially in the fields of pheochromocytoma (pheochromocytoma) and paraganglioma (paraganglioma, referred to as PPGL).

1. Application of besetifan in VHL syndrome-related tumors

VHL syndrome is a rare hereditary tumor syndrome. Patients are susceptible to a variety of tumors, including renal clear cell carcinoma, central nervous system hemangioblastoma, and pancreatic neuroendocrine tumors. Bestifan blocks the adaptive response of tumor cells to hypoxia by inhibiting HIF-2α, thereby inhibiting tumor growth. According to clinical trial results updated in 2025, Bestifan has shown good efficacy and tolerability in the treatment of VHL-related tumors. Some patients' tumors have stabilized or shrunk, and their quality of life has improved.

2. The efficacy of besetifan in pheochromocytoma and paraganglioma

Pheochromocytoma and paraganglioma are rare tumors originating from the adrenal medulla or paraganglion and are often accompanied by symptoms such as hypertension and tachycardia. Bestifan reduces the ability of tumor cells to adapt to hypoxia and inhibits tumor growth by inhibiting HIF-2α. In 2025Maymonth, the U.S. FDA approved bezutivan for the treatment of locally advanced, unresectable or metastatic pheochromocytoma and paraganglioma, becoming the first approved oral targeted therapy drug. Clinical studies have shown that bestivan has achieved significant efficacy in these patients, with some patients having tumors stabilized or shrunk and symptoms improved.

3. Research progress of besetifan in other rare tumors

ExceptIn addition to VHL related tumors and pheochromocytoma, the application of besetivan in other rare tumors is also constantly being explored. For example, in some types of lung cancer, liver cancer, and pancreatic cancer, bezutivan may inhibit tumor growth and metastasis by inhibiting HIF-2α. Although these studies are still in their early stages, preliminary results suggest that bezutivan may have potential therapeutic value in a variety of tumor types.

4. Clinical Application Prospects of Bestifan

Besetifan, as the firstHIF-2α inhibitor, provides a new option for the treatment of a variety of rare tumors. With the deepening of clinical research, the efficacy and safety of besetifan in more rare tumors will be further verified. In the future, besetifan may become an important drug for the treatment of these rare tumors and improve patients' prognosis and quality of life. However, since the indications and efficacy of bestifan are still expanding, patients should fully communicate with professional doctors before use to develop an individualized treatment plan.

In summary, the clinical application and efficacy research of besetifan in a variety of rare tumors have made significant progress, providing patients with new treatment options. With the deepening of research, bezutivan is expected to play an important role in the treatment of more rare tumors.

Reference materials:https://www.drugs.com/