Midostaurin (3) – Rydapt®
Systemic Mastocytosis
Characteristics
| Start date | 15.11.2023 – Marketing authorisation: 18.09.2017 |
|---|---|
| Resolution | 02.05.2024 |
| INN | Midostaurin |
| Brand name | Rydapt® |
| Pharm. company | Novartis Pharma AG |
| G-BA Procedure ID | D-992 |
| ATC code | L01EX10 Other protein kinase inhibitors (L01EX) |
| ICD-10 codes (AIS) | C94.30Mast cell leukemia with failed remission, C94.31Mast cell leukemia, in remission, C96.2Malignant mast cell neoplasm |
| Alpha-ID codes (AIS) | I116195Aggressive systemic mastocytosis, I18159Mast cell leukemia, I31132Mast cell leukemia in complete remission |
| ORPHAcodes (AIS) | 98850Aggressive systemic mastocytosis, 98851Mast cell leukemia, 98851Mast cell leukemia in complete remission |
| Therapeutic area | Oncological diseases Mastocytosis / Systemic mastocytosis Orphan (turnover limit) |
| Reason for procedure |
Reassessment: Orphan turnover exceeded
Original resolution: Midostaurin (1) (05.04.2018) |
| Specialty | Bundling ACT change |
| Therapeutic indication of the resolution |
|---|
|
Rydapt is used as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematologic neoplasia (SM-AHN) or mast cell leukemia (MCL) |
| Subpopulation | Indication | Comparator |
|---|---|---|
| Adults with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematologic neoplasia (SM-AHN) or mast cell leukemia (MCL) | Patient-specific therapy with selection of – Avapritinib (only for people after at least one prior systemic therapy and with platelet counts ≥ 50 x 109/L), – cladribine and – Imatinib (only for people without KIT D816V mutation or with unknown KIT mutation status and for people with existing eosinophilia with FIP1L1-PDGFRA fusion gene), taking into account the general condition, KIT mutation status and previous therapy |
Studies and Results
|
No. of studies
(best subpopulation) |
0 (Data not accepted) |
|---|---|
|
Study design
(best subpopulation) |
Data not accepted (Dossier: Single-arm + historical comparison) |
| Reason for dividing into subpopulations (G-BA) | Time of treatment |
| ACT change | 28.11.2023 – Neue Therapiestandards |
- Clinical trials
- The CPKC412D2201 and CPKC412A2213 trials are single-arm Phase II trials.
- The CPKC412D2201 trial enrolled 116 patients with aggressive systemic mastocytosis or mast cell leukaemia.
- The CPKC412A2213 trial included 26 patients with histologically confirmed ASM or MCL.
Adults with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated haematological neoplasia (SM-AHN) or mast cell leukaemia (MCL)
- An additional benefit is not proven.
- Consequently, there are no suitable data available overall for assessing the additional benefit of midostaurin.
- Consequently, no additional benefit of midostaurin has been demonstrated for adults with aggressive systemic mastocytosis (ASM), There is no proof that systemic mastocytosis with associated haematological neoplasia (SM-AHN) or mast cell leukaemia (MCL) compared with the appropriate comparator therapy is better than the comparator therapy.
- Overall assessment
- The results of the submitted single-arm studies CPK412D2201 and CPK412A2213 are not suitable for assessing additional benefit, as they do not allow for a comparison with the appropriate comparator therapy.
- The results of Lübke’s non-randomised comparison are also not taken into account for the benefit assessment due to the lack of detailed information on methodology and the failure to implement the appropriate comparator therapy.
Courtesy translation only, please refer to the German original.
Associated procedures
| Midostaurin (3) | Rydapt® | Novartis Pharma AG | Systemic Mastocytosis | 300–400 | 100% additional benefit not proven Orphan (turnover limit) | |
| Midostaurin (2) | Rydapt® | Novartis Pharma AG | Acute myeloic Leukemia, FLT3-Mutation | 380–1,040 | 100% additional benefit not proven Orphan (turnover limit) | |
| Midostaurin (1) | Rydapt® | Novartis Pharma GmbH | Acute myeloid leukaemia (AML); Systemic mastocytosis (ASM) |
0
400–710 |
80% considerable additional benefit Orphan repealed |
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