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| Molecular Profile | VHL inact mut |
| Therapy | Belzutifan |
| Indication/Tumor Type | hemangioblastoma |
| Response Type | sensitive |
| Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
|---|---|---|---|---|---|---|---|
| VHL inact mut | hemangioblastoma | sensitive | Belzutifan | FDA approved | Actionable | In a Phase II trial (LITESPARK-004) that supported FDA approval, Welireg (belzutifan) treatment was safe and resulted in an objective response rate (ORR) of 44% (22/50) in patients with Von Hippel-Lindau disease-associated CNS hemangioblastoma harboring germline VHL mutations, ORR was 76% (19/25) in those with measurable baseline lesions (PMID: 39284337; NCT03401788). | 39284337 detail... |
| VHL inact mut | hemangioblastoma | sensitive | Belzutifan | Guideline | Actionable | Welireg (belzutifan) is included in guidelines for patients with Von Hippel-Lindau disease-associated CNS hemangioblastoma that do not require immediate surgery (NCCN.org). | detail... |
| PubMed Id | Reference Title | Details |
|---|---|---|
| NCCN.org | Full reference... | |
| Welireg (belzutifan) FDA Drug Label | Full reference... | |
| (39284337) | Belzutifan for patients with von Hippel-Lindau disease-associated CNS haemangioblastomas (LITESPARK-004): a multicentre, single-arm, phase 2 study. | Full reference... |