The Food and Drug Administration (FDA) has approved MK-6482 (belzutifan) for adult patients with von Hippel-Lindau disease who require treatment for associated renal cell carcinoma, central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET), not requiring immediate surgery.

Von Hippel-Lindau disease is a genetic disease characterized by the formation of tumors and cysts in many different parts of the body. The condition can result in a variety of noncancerous or cancerous tumors due to mutations in the VHL gene. People with von Hippel-Lindau disease are also at increased risk for developing other cancers, including renal cell carcinoma, CNS, hemangioblastomas, pNETs.

The approval was based on findings from the ongoing, open-label, single-arm phase 2 study. The primary endpoint of the study was the objective response rate in kidney masses. This study observed that 36% of patients had confirmed responses with an additional 11% of patients having unconfirmed, partial responses. Furthermore, there were objective responses seen in some patients’ pancreatic lesions as well as stabilization or improvement in patients’ hemangioblastomas.

In an exclusive interview with CheckRare, Eric Jonasch, MD, Professor, Department of Genitourinary Medical Oncology at MD Anderson Cancer Center talked about the phase 2 study that led to the drug’s approval.



As Dr. Jonasch says in the above video, the psychological burden of von Hippel-Lindau disease on patients is tremendous. Therefore, this approval is of great importance for this patient population.

For more information about Von Hippel-Lindau disease and other rare cancers, visit 


Reference Material

U.S. Food and Drug Administration. FDA approves belzutifan for cancers associated with von Hippel-Lindau disease. [Press Release].