WELIREG belzutifan

Drug Class

Hypoxia-inducible factor 2 alpha (HIF-2α) inhibitor

Mechanism

Inhibits HIF-2α transcription factor binding to its partner ARNT, reducing the expression of genes involved in cell proliferation, angiogenesis, and tumor growth under hypoxic conditions.

Approved Indication

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.

What This Means For Patients

Welireg (belzutifan) is a first-in-class HIF-2α inhibitor approved by the FDA, NMPA, and EMA. Specifically developed for patients with von Hippel-Lindau (VHL) disease, it targets the molecular pathways driving tumor growth in VHL-associated kidney and brain cancers. Under special import regulations, patients can access this therapy in the Boao Lecheng pilot zone.

Regulatory Approvals

• FDA — approved (2021) — label
• NMPA — approved (2024) — source
• EMA — approved (2025) — source

Pivotal Clinical Evidence

• LITESPARK-004 — Jonasch et al., N Engl J Med 2021 (NCT03401788) — DOI

Sources

• https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/215383s000lbl.pdf
• https://www.nmpa.gov.cn/