Study
| Single-arm, phase 2 trial (LITESPARK-004) |
| VHL disease patients with RCC, CNS hemangioblastomas, or pancreatic NETs |
| Belzutifan 120 mg orally once daily |
Efficacy
| ORR: 67% (11% CR, 56% PR) (RCC) |
| mPFS: 49.8 mos (95% CI: 49.8–NR) (RCC) |
| ORR: 48% (8% CR) (CNS hemangioblastomas) |
| mPFS: NR (CNS hemangioblastomas) |
| ORR: 91% (50% CR) (pancreatic NETs) |
| mPFS: NR (pancreatic NETs) |
| ORR: 67% (11% CR) (RCC) |
| Improvement in retinal hemangioblastomas: 93% of pts, 94% of eyes |
Safety
| Grade ≥3 AEs: anaemia (11%), fatigue (5%), urinary tract infection (2%), hypoxia (2%), blister (2%) |
| Serious TRAEs: anaemia, UTI, intracranial hemorrhage, hypoxia (4 pts) |
Lancet Oncol 2025;26: 571–82
http://doi.org/10.1016/S1470-2045(24)00704-4
Reviewed by Ulas D. Bayraktar, MD on May 4, 2025
