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New Drug: Belzutifan for cancers associated with von Hippel-Lindau disease

New Drug: Belzutifan for cancers associated with von Hippel-Lindau disease
Study:
  • Open label Phase II trial (Study 004)
  • Adults with germline vHL alterations with clear-cell RCC (N=61). Pts had other vHL-associated tumors: 12 pts had measurable pNET, 24 pts had measurable CNS hemangioblastoma
  • Belzutifan 120 mg PO daily
Efficacy:
  • ORR: 49% (ccRCC), 83% (pNET), 63% (CNS hemangioblastoma)
  • 56% of responders with ccRCC had DoR over one year
Safety:
  • All pts had AEs. No Grade 4 AEs reported. Most common AE was anemia (90%) with grade 3 anemia in 7%

J Clin Oncol 2021;39: suppl 4555.

Srinivasan R, et al. Phase 2 study of belzutifan (MK-6482), an oral hypoxia-inducible factor 2α (HIF-2α) inhibitor, for Von Hippel-Lindau (VHL) disease-associated clear cell renal cell carcinoma (ccRCC)

https://doi.org/10.1200/JCO.2021.39.15_suppl.4555

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