Belzutifan is a novel HIF-2 α inhibitor that provides a new targeted therapy option for patients with inoperable VHL related tumors. This drug significantly delays tumor progression by specifically inhibiting the hypoxia inducible factor pathway, and has good safety.

1. Mechanism of action

Beizu Tifan selectively binds to HIF-2 α protein, blocking its dimerization with HIF-1 β and inhibiting the transcription of downstream pro angiogenic and cell proliferation genes. This mechanism is particularly applicable to the abnormal accumulation of HIF-2 α caused by VHL gene deletion.

2. Indications

(1) VHL associated renal cell carcinoma: Treatment for adult patients with VHL associated clear cell renal cell carcinoma who do not require immediate surgery.

(2) VHL related pancreatic neuroendocrine tumors: used to control the growth of pancreatic neuroendocrine tumors associated with VHL syndrome.

(3) VHL related central nervous system hemangioblastoma: suitable for VHL related hemangioblastoma that cannot be surgically removed.

3. Clinical advantages

(1) The objective remission rate reached 49%, and the median duration of remission was not achieved.

(2) 96% of renal cell carcinoma patients achieve tumor shrinkage, and the shrinkage rate of pancreatic neuroendocrine tumors is 82%.

(3) The most common adverse event is anemia (90%), but it is mostly grade 1-2.

4. Medication plan

The recommended dosage is 120mg orally once daily, taken on an empty stomach or after meals. Medication should be continued until disease progression or intolerable toxicity occurs, and the entire tablet should be swallowed during administration.

5. Special populations

(1) Pregnancy: May cause fetal damage, contraception should be used until one week after the last dose.

(2) Breastfeeding period: It is recommended to stop taking medication or breastfeeding.

(3) Liver injury: Mild without adjustment, lack of data for moderate to severe cases.