Belzutifan (2) – Welireg®
Von Hippel-Lindau syndrome (VHL)-associated tumours
Characteristics
| Start date | 01.04.2025 – Marketing authorisation: 12.02.2025 |
|---|---|
| Resolution | 18.09.2025 |
| INN | Belzutifan |
| Brand name | Welireg® |
| Pharm. company | MSD Sharp & Dohme GmbH |
| G-BA Procedure ID | D-1175 |
| ATC code | L01XX74 Other antineoplastic agents (L01XX) |
| ICD-10 codes (AIS) | Q85.8Other phakomatoses, not elsewhere classified |
| Alpha-ID codes (AIS) | I79461Von Hippel-Lindau syndrome |
| Therapeutic area | Oncological diseases Neuroendocrine tumors (NET), Von Hippel-Lindau syndrome (VHL)-associated tumors |
| Reason for procedure | Initial assessment |
| Regulatory status | Conditional Approval |
| Specialty | Bundling |
| Therapeutic indication of the resolution |
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|
Welireg is indicated as monotherapy for the treatment of von Hippel-Lindau syndrome in adults who require therapy for associated local renal cell carcinoma (RCC), haemangioblastoma of the central nervous system (CNS) or pancreatic neuroendocrine tumours (pNET) and for whom local therapies are unsuitable. |
| Subpopulation | Indication | Comparator |
|---|---|---|
| Adults with renal cell carcinoma (RCC) associated with von Hippel-Lindau syndrome, haemangioblastoma of the central nervous system (CNS) or pancreatic neuroendocrine tumour (pNET) who are not eligible for local therapies and who require therapy | Observational waiting |
Studies and Results
|
No. of studies
(best subpopulation) |
1 (LITESPARK 004) 0 (Data not accepted) |
|---|---|
|
Study design
(best subpopulation) |
Data not accepted (Dossier: Single-arm + historical comparison) |
- Clinical trials
- The ongoing, open-label, single-arm LITESPARK 004 trial is the pivotal Phase II trial on the basis of which belzutifan was granted conditional approval.
- The Von Hippel-Lindau Natural History Study is a retrospective, non-interventional study based on data from the National Cancer Institute Urologic Oncology Branch Von Hippel-Lindau Hereditary Database.
Adults with renal cell carcinoma (RCC) associated with von Hippel-Lindau syndrome, central nervous system (CNS) haemangioblastoma or pancreatic neuroendocrine tumour (pNET) associated with von Hippel-Lindau syndrome, for whom local therapies are not an option and who require treatment
- An additional benefit is not proven.
- Overall, the data presented are not sufficient to demonstrate any additional benefit of belzutifan compared with the appropriate comparator therapy; therefore, there is no additional benefit of belzutifan for the treatment of adults with renal cell carcinoma (RCC), central nervous system (CNS) haemangioblastoma or pancreatic neuroendocrine tumour (pNET), for whom local therapies are not an option and who require treatment, is not proven.
- Conclusion
- Overall, the data presented are not sufficient to demonstrate any additional benefit of belzutifan compared with the appropriate comparator therapy; therefore, no additional benefit of belzutifan has been demonstrated for the treatment of adults with renal cell carcinoma associated with von Hippel–Lindau syndrome (RCC), central nervous system (CNS) haemangioblastoma or pancreatic neuroendocrine tumour (pNET), for whom local therapies are not an option and who require treatment, is not proven.
Courtesy translation only, please refer to the German original.
Associated procedures
| Belzutifan (2) | Welireg® | MSD Sharp & Dohme GmbH | Von Hippel-Lindau syndrome (VHL)-associated tumours | 80–970 | 100% additional benefit not proven | |
| Belzutifan (1) | Welireg® | MSD Sharp & Dohme GmbH | Renal cell carcinoma, advanced, after ≥ 2 prior therapies | 65–940 | 50% Hint for minor additional benefit |
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