Avapritinib (5) – Ayvakyt®
Advanced systemic mastocytosis, following at least one prior course of treatment
Characteristics
| Start date | 01.11.2025 – Marketing authorisation: 24.03.2022 |
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| Resolution | 16.04.2026 |
| INN | Avapritinib |
| Brand name | Ayvakyt® |
| Pharm. company | Blueprint Medicines (Germany) GmbH |
| G-BA Procedure ID | D-1262 |
| ATC code | L01EX18 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 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 aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated haematological neoplasia (SM-AHN) or mast cell leukaemia (MCL) following at least one course of systemic therapy. |
| Subpopulation | Indication | Comparator |
|---|---|---|
| Erwachsene mit aggressiver systemischer Mastozytose (ASM), systemischer Mastozytose mit assoziierter hämatologischer Neoplasie (SM-AHN) oder Mastzellleukämie (MCL) nach zumindest einer systemischen Therapie |
Studies and Results
- Clinical trials
- The PATHFINDER trial is an open-label, multicentre, single-arm Phase II trial of avapritinib for the treatment of adults with advanced systemic mastocytosis.
- The EXPLORER trial is an open-label, multicentre, single-arm Phase I trial of avapritinib for the treatment of adult patients with advanced systemic mastocytosis, comprising a dose-escalation phase (Part 1) and an extension phase (Part 2).
Adults with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated haematological neoplasia (SM-AHN) or mast cell leukaemia (MCL) who have received at least one course of systemic therapy
- An additional benefit is not proven.
- The single-arm studies PATHFINDER and EXPLORER submitted by the pharmaceutical manufacturer do not allow for a comparison with the appropriate comparator therapy. Consequently, there are no data available to enable an assessment of the additional benefit.
Courtesy translation only, please refer to the German original.
Associated procedures
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