3
Indication details
- Control Arm
- Single arm
- Therapeutic Indication
- EMA: Belzutifan as monotherapy for the treatment of adult patients with von Hippel-Lindau disease who require therapy for associated, localised renal cell carcinoma (RCC), central nervous system (CNS) haemangioblastomas, or pancreatic neuroendocrine tumours (pNET), and for whom localised procedures are unsuitable. FDA: For adult patients with von Hippel-Lindau (VHL) disease who require therapy for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET), not requiring immediate surgery.
- Tumour Type
-
Genitourinary Cancers
- Tumour Sub-type
- von Hippel-Lindau disease-associated Renal Cell Carcinoma
- Tumour Stage
- Early
- Trial Name
- MK-6482 (Study 004)
- NCT Number
- NCT03401788
- Trial Phase
- Phase II
Approval details
- FDA Approval
- FDA approval August 2021
- EMA Approval
- EMA (CHMP) December 2024. Pending EC decision
Primary Outcome(s)
- Primary Outcome(s)
- ORR, DoR
- Evaluated Outcome
- ORR, DoR
- Form(s)
- Form 3
Outcome Data
- ORR
- 49% (36%-62%)
- DoR
- 9+ months
Adjustments
- QoL Comment
-
Not qualified for an ESMO-MCBS credit
Score (after adjustments)
- Preliminary non-curative score
-
3
- Non-curative score
-
3
Scorecard details
- ESMO-MCBS version
- ESMO-MCBS v1.1
- Scorecard ID
- 314
- Scorecard version
- 1
- Issue date
- 20.01.2022
- Last update
- 09.01.2024
Legend
Information about symbols, abbrevations and color codes
Red = negative
Green = positive
Grey = neutral
?
Grey + ? = neutral with pending data
- DFS
- Disease-Free Survival
- DoR
- Duration of Response
- EFS
- Event-Free Survival
- HR
- Hazard Ratio
- NEB
- No evaluable benefit
- NI
- Non-inferiority Study
- ORR
- Overall Response Rate
- OS
- Overall Surival
- pCR
- Pathological Complete Response/Remission
- PFS
- Progression-Free Survival
- QoL
- Quality of Life
- RFS
- Relapse-Free Survival
- RR
- Response Rate
QoL adjustments
Other adjustments*
Serious and disabling adverse effects
Red = negative / deterioration
Green = positive / improvement
QoL adjustments
Red = negative / deterioration
Green = positive / improvement
Other adjustments*
Red = negative / deterioration
Green = positive / improvement
Serious and disabling adverse effects
Red = negative / deterioration
Green = positive / improvement
* Other adjustments include:
- Long-term plateau in the survival curve
- Long-term plateau in the PFS curve
- Early stopping or crossover
- Only improved PFS mature data shows no OS advantage and no improved Qol.