3
ESMO-MCBS v1.1
Scorecard version: 1
Indication details
- Tumour Type
- Endocrine Tumours
- Tumour Sub-type
- Thyroid Cancer
- Tumour Sub-Group
- RET-mutant
- Tumour stage
- Advanced or metastatic
- Control Arm
- Single arm (Phase I/II)
- Treatment Setting
- As monotherapy for the treatment of adult and adolescents ≥12 years of age with advanced RET-mutant medullary thyroid cancer (MTC)
- Trial Name
- LIBRETTO-001 (Cohort 4)
Primary Outcome(s)
- Primary Outcome(s)
- ORR
- Evaluated Outcome
- ORR/DoR
- Form(s)
- Form 3
Outcome Data
- ORR
- 73%
- DoR
- 22.0 months
Final Score (after adjustments)
- Preliminary non-curative score
- 3
- Final non-curative Score
- 3
- Comment
- EMA (CHMP) December 2020 EC decision February 2021
EMA (CHMP) extension of indication July 2022 EC decision September 2022
FDA approval May 2020 - Issue date
- 4.11.2020
- Release date
- 4.11.2020
- Last update
- 25.07.2022
DFS, disease-free survival; DoR, duration of response; EFS, event-free survival; FFS, failure-free survival; HR, hazard ratio; iDFS, invasive disease-free survival; ITT, intention to treat; MFS, metastasis-free survival; ORR, overall response rate; OS, overall survival; pCR, pathologic complete response/remission; PE, point estimate; PFS, progression-free survival; QoL, quality of life; RFS, relapse-free survival; RR, response rate; SD, stable disease; SSA, somatostatin analogue; SSTR, somatostatin receptor; TKI, tyrosine kinase inhibitor, TTP, time to progression