Avapritinib (6) – Ayvakyt®
Gastrointestinal stromal tumours
Characteristics
| Start date | 01.11.2025 – Marketing authorisation: 24.09.2020 |
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| Resolution | 16.04.2026 |
| INN | Avapritinib |
| Brand name | Ayvakyt® |
| Pharm. company | Blueprint Medicines (Germany) GmbH |
| G-BA Procedure ID | D-1263 |
| ATC code | L01EX18 Other protein kinase inhibitors (L01EX) |
| ICD-10 codes (AIS) | C15.9Malignant neoplasm of esophagus, unspecified, C16.9Gastric cancer NOS, C17.9Malignant neoplasm of small intestine, unspecified, C18.9Malignant neoplasm of large intestine NOS, C26.9Malignant neoplasm of ill-defined sites within the digestive system, C48.2Malignant neoplasm of peritoneum, unspecified |
| Alpha-ID codes (AIS) | I117054Gastrointestinal stromal tumor, I134833GIST (gastrointestinal stromal tumor) of the small intestine, I134850GIST (gastrointestinal stromal tumor) of the esophagus, I134868GIST (gastrointestinal stromal tumor) of the stomach, I134898GIST (gastrointestinal stromal tumor) of the colon, I134908GIST (gastrointestinal stromal tumor) of the peritoneum |
| ORPHAcodes (AIS) | 44890Gastrointestinal stromal tumor, 44890GIST (gastrointestinal stromal tumor) of the small intestine, 44890GIST (gastrointestinal stromal tumor) of the esophagus, 44890GIST (gastrointestinal stromal tumor) of the stomach, 44890GIST (gastrointestinal stromal tumor) of the colon, |
| Therapeutic area | Oncological diseases Orphan (turnover limit) |
| Reason for procedure | Reassessment: Orphan turnover exceeded |
| Therapeutic indication of the resolution |
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Ayvakyt is indicated as monotherapy for the treatment of adult patients with inoperable or metastatic gastrointestinal stromal tumours (GIST) harbouring the platelet-derived growth factor receptor alpha (PDGFRA) PDGFRA) D842V mutation. |
| Subpopulation | Indication | Comparator |
|---|---|---|
| Erwachsene Patientinnen und Patienten mit inoperablen oder metastasierten gastrointestinalen Stromatumoren (GIST), die die Thrombozyten-Wachstumsfaktor-Rezeptoralpha (PDGFRA)-D842V-Mutation aufweisen |
Studies and Results
- Clinical trials
- The NAVIGATOR study is a single-arm, multicentre Phase I study of avapritinib, divided into a dose-escalation phase (Part 1) and an expansion phase (Part 2).
- The CS3007-101 trial is a non-randomised Phase I/II trial of avapritinib, conducted exclusively in China.
Adult patients with inoperable or metastatic gastrointestinal stromal tumours (GIST) harbouring the platelet-derived growth factor receptor alpha (PDGFRA) D842V mutation
- The additional benefit is not proven.
- Consequently, there are no suitable data available overall for assessing the additional benefit of avapritinib. Consequently, no additional benefit of avapritinib has been demonstrated for patients with inoperable or metastatic gastrointestinal stromal tumours (GIST) who carry the platelet-derived growth factor receptor alpha (PDGFRA)-D842V mutation, compared with the appropriate comparator therapy, does not provide proof.
- mortality
- The single-arm studies NAVIGATOR and CS3007-101 submitted by the pharmaceutical manufacturer do not allow for a comparison with the appropriate comparator therapy and are therefore not suitable for assessing the additional benefit of avapritinib as monotherapy.
- morbidity
- The single-arm studies NAVIGATOR and CS3007-101 submitted by the pharmaceutical manufacturer do not allow for a comparison with the appropriate comparator therapy and are therefore not suitable for assessing the additional benefit of avapritinib as monotherapy.
- Side effects
- The single-arm studies NAVIGATOR and CS3007-101 submitted by the pharmaceutical manufacturer do not allow for a comparison with the appropriate comparator therapy and are therefore not suitable for assessing the additional benefit of avapritinib as monotherapy.
- Conclusion
- The single-arm studies NAVIGATOR and CS3007-101 submitted by the pharmaceutical manufacturer do not allow for a comparison with the appropriate comparator therapy and are therefore not suitable for assessing the additional benefit of avapritinib as monotherapy.
- The results of the VOYAGER study, which were already submitted in the dossier for the initial assessment and are presented comparatively in the results section, a propensity score (PS)-adjusted indirect comparison with the NAVIGATOR and BLU-285-1002 studies and the retrospective study by Cassier et al, 2012, were not analysed in the present procedure and are therefore not suitable for assessing additional benefit.
- Consequently, there are no suitable data available overall for the assessment of the additional benefit of avapritinib.
Courtesy translation only, please refer to the German original.
Associated procedures
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