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    DRUG:

    Ayvakit (avapritinib)

    i
    Other names: CS-3007, CS 3007, BLU-285, BLU 285, BLU285, CS3007
    Company:
    CStone Pharma, Sanofi
    Drug class:
    PDGFR antagonist, mutant c-KIT inhibitor
    Related drugs:
    2d
    Deep Learning Predicts Mutations and Outcomes in Gastrointestinal Stromal Tumors from Whole-Slide Images. (PubMed, Cancer Res)
    For therapeutic categories, performance reached 0.84 for avapritinib sensitivity and 0.81 for imatinib sensitivity. Prognostic performance was comparable to pathology-based scores, with highest discrimination in the overall cohort and in patients without adjuvant therapy. DL applied to WSIs enables prediction of molecular alterations, treatment sensitivity, and RFS in GIST, performing comparably to established risk scores across international cohorts, providing a baseline for future multimodal predictors.
    Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
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    KIT mutation • PDGFRA D842V • PDGFRA mutation
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    imatinib • Ayvakit (avapritinib)
    11d
    Microglia in diffuse midline glioma contribute to extracellular matrix remodelling and cancer cell invasion. (PubMed, Cell Death Dis)
    Functional invasion assays using a panel of patient-derived DMG, H3K27M cells, revealed that microglia-derived fibronectin significantly enhances tumour cell invasiveness, while its chemical inhibition with RGDS peptide or Avapritinib or its genetic silencing using small-interfering RNAs effectively suppresses invasion. Across independent patient cohorts (Kids First, PNOC, and CBTTC), and in archival tissues, DMG tumours were found to exhibit elevated expression of ECM components, and high fibronectin expression that correlated with poor prognosis. These findings suggest that microglia actively contribute to DMG invasiveness through ECM component production, identifying fibronectin as a potential therapeutic target in this lethal paediatric cancer.
    Journal
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    FN1 (Fibronectin 1)
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    Ayvakit (avapritinib)
    20d
    Sustainable high-throughput microwell spectrophotometric methods for avapritinib quality control via charge-transfer complexation. (PubMed, Sci Rep)
    A comprehensive multi-metric sustainability assessment-utilizing ten tools including AES, AGREE, BAGI, RGB, and RAPI-confirmed the methods' excellent greenness (e.g., AES score = 90), superior practicality, and robust analytical performance, achieving a high White Index of 94.2%. With a substantially higher throughput of ~ 500 samples/hour and minimal solvent consumption (200µL/well), the proposed MW-SPMs offers a rapid, sustainable, and cost-effective alternative to conventional chromatographic and spectrofluorimetric techniques for routine pharmaceutical analysis, aligning with multiple United Nations Sustainable Development Goals.
    Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
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    PDGFRA D842V • PDGFRA mutation
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    Ayvakit (avapritinib)
    21d
    Efficacy and safety analysis of avapritinib in children with RUNX1::RUNX1T1 positive-acute myeloid leukemia with KIT mutations (PubMed, Zhonghua Xue Ye Xue Za Zhi)
    Avapritinib provides a favorable initial response in children with RUNX1::RUNX1T1-positive AML harboring KIT mutations, enabling deeper molecular remission; however, response rates decline with prolonged treatment. Avapritinib has a favorable safety profile.
    Retrospective data • Journal
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    KIT (KIT proto-oncogene, receptor tyrosine kinase) • RUNX1 (RUNX Family Transcription Factor 1) • RUNX1T1 (RUNX1 Partner Transcriptional Co-Repressor 1)
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    KIT mutation
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    Ayvakit (avapritinib)
    21d
    Disrupting MED8-dependent epigenetic reprogramming augments avapritinib sensitivity in PDGFRA-driven glioma. (PubMed, J Exp Clin Cancer Res)
    This study delineates a novel MED8-SE-PDGFRA epigenetic axis driving resistance. The combination of avapritinib and venetoclax, co-targeting the oncogenic signal and its transcriptional regulator, presents a translatable dual-targeting strategy to improve outcomes in PDGFRA-driven glioma.
    Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • CDK7 (Cyclin Dependent Kinase 7)
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    Venclexta (venetoclax) • Ayvakit (avapritinib)
    2ms
    MegaMOST: A Study Evaluating the Activity of Anti-cancer Treatments Targeting Tumor Molecular Alterations/Characteristics in Advanced / Metastatic Tumors. (clinicaltrials.gov)
    P2, N=455, Recruiting, Centre Leon Berard | Trial completion date: Nov 2026 --> Oct 2027 | Trial primary completion date: Feb 2026 --> Oct 2026
    Trial completion date • Trial primary completion date
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    KRAS (KRAS proto-oncogene GTPase) • BRAF (B-raf proto-oncogene) • ALK (Anaplastic lymphoma kinase) • FLT3 (Fms-related tyrosine kinase 3) • MET (MET proto-oncogene, receptor tyrosine kinase) • NRAS (Neuroblastoma RAS viral oncogene homolog) • KIT (KIT proto-oncogene, receptor tyrosine kinase) • ROS1 (Proto-Oncogene Tyrosine-Protein Kinase ROS) • NTRK2 (Neurotrophic tyrosine kinase, receptor, type 2) • HRAS (Harvey rat sarcoma viral oncogene homolog) • CDKN2A (Cyclin Dependent Kinase Inhibitor 2A) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • NF1 (Neurofibromin 1) • AXL (AXL Receptor Tyrosine Kinase) • CCND1 (Cyclin D1) • CDK4 (Cyclin-dependent kinase 4) • PTPN11 (Protein Tyrosine Phosphatase Non-Receptor Type 11) • SMAD4 (SMAD family member 4) • RAF1 (Raf-1 Proto-Oncogene Serine/Threonine Kinase) • FLT1 (Fms-related tyrosine kinase 1) • CDK6 (Cyclin-dependent kinase 6) • CCND3 (Cyclin D3) • TYRO3 (TYRO3 Protein Tyrosine Kinase) • NTRK (Neurotrophic receptor tyrosine kinase)
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    BRAF mutation • NRAS mutation • BRAF V600 • KIT mutation • CDKN2A deletion • HRAS mutation • KRAS G12 • PDGFRA mutation • KRAS amplification • NRAS G12
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    Mekinist (trametinib) • Tafinlar (dabrafenib) • Alecensa (alectinib) • Cabometyx (cabozantinib tablet) • Stivarga (regorafenib) • Kisqali (ribociclib) • Ayvakit (avapritinib) • siremadlin (HDM201)
    4ms
    Gastrointestinal Stromal Tumors: Molecular Mechanisms of Drug Resistance and Advances in Therapeutic Strategies. (PubMed, J Gastroenterol Hepatol)
    The introduction of imatinib, a selective tyrosine kinase inhibitor (TKI), revolutionized the management of advanced GIST...Current approaches emphasize molecular subtype-guided first-line therapy, ctDNA-driven sequencing of subsequent lines, and the use of novel TKIs (e.g., avapritinib, ripretinib, bezuclastinib) tailored to specific resistance mutations. Furthermore, we explore emerging modalities such as boron neutron capture therapy (BNCT), which offers a kinase-independent mechanism to target resistant disease, and combination strategies that integrate immunotherapy, epigenetic modulators, and pathway-specific inhibitors. Advances in liquid biopsy and molecular profiling are enabling real-time adaptation of therapy, moving GIST management toward a dynamic, personalized paradigm aimed at overcoming resistance and improving patient outcomes.
    Review • Journal • IO biomarker
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    KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
    |
    KIT mutation • PDGFRA mutation
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    imatinib • Ayvakit (avapritinib) • Qinlock (ripretinib) • bezuclastinib (PLX9486)
    4ms
    Prediction of Mutations and Outcome in Gastrointestinal Stromal Tumors with Deep Learning: A Multicenter, Multinational Study. (PubMed, medRxiv)
    For therapeutic categories, performance reached 0.84 for avapritinib sensitivity, 0.81 for imatinib sensitivity. DL applied to WSIs enables prediction of molecular alterations, treatment sensitivity, and RFS in GIST, performing comparably to established risk scores across international cohorts, providing a baseline for future multimodal predictors. Deep learning on histology predicts KIT and PDGFRA mutations in a large international cohort of GISTs from multiple centers Whole-slide image models stratify recurrence-free survival comparable to pathology-based risk scores Prognostic value of deep learning is preserved in adjuvant therapy subgroups, supporting treatment duration decisions.
    Journal
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    PDGFRA (Platelet Derived Growth Factor Receptor Alpha)
    |
    KIT mutation • PDGFRA D842V • PDGFRA mutation
    |
    imatinib • Ayvakit (avapritinib)
    4ms
    Gastrointestinal Stromal Tumors: Histopathological Spectrum, Molecular Subtypes, and Implications for Targeted Therapy. (PubMed, Cureus)
    Targeted tyrosine kinase inhibitors (TKIs) have transformed GIST management, with imatinib as the foundational first-line therapy and subsequent agents, sunitinib, regorafenib, avapritinib, and ripretinib, addressing primary or secondary resistance driven by diverse mutational patterns. Emerging therapeutic directions include next-generation kinase inhibitors, heat shock protein inhibitors, immunotherapy, metabolic and epigenetic targeting, and biomarker-driven individualized treatment strategies. This review synthesizes contemporary advances in the histopathological, molecular, and therapeutic landscape of GISTs, emphasizing an integrated diagnostic approach and highlighting ongoing efforts to overcome therapeutic resistance and optimize personalized care.
    Review • Journal • IO biomarker
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    KRAS (KRAS proto-oncogene GTPase) • BRAF (B-raf proto-oncogene) • KIT (KIT proto-oncogene, receptor tyrosine kinase) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • NF1 (Neurofibromin 1) • CD34 (CD34 molecule) • NTRK (Neurotrophic receptor tyrosine kinase) • ANO1 (Anoctamin 1)
    |
    KRAS mutation • BRAF mutation • KIT mutation • PDGFRA D842V • PDGFRA mutation • NTRK fusion
    |
    imatinib • sunitinib • Stivarga (regorafenib) • Ayvakit (avapritinib) • Qinlock (ripretinib)
    4ms
    Efficacy and safety of avapritinib in advanced systemic mastocytosis: 4-year follow-up of the PATHFINDER study. (PubMed, Blood Adv)
    Eleven (10%) patients experienced TEAEs leading to death, of which 1 was deemed related to avapritinib by the principal investigator. With 4-year follow-up, avapritinib-treated patients with AdvSM experienced deep and durable responses and a favorable benefit-risk profile.
    Journal
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    KIT (KIT proto-oncogene, receptor tyrosine kinase)
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    Ayvakit (avapritinib)
    5ms
    Case Report: A rare case of synchronous ovarian mixed germ cell tumor and mast cell leukemia in a pediatric patient. (PubMed, Front Oncol)
    Targeted therapy with avapritinib and ruxolitinib was initiated but yielded limited response. Given the consistent co-occurrence of KIT mutations in previously reported similar cases, we propose the recognition of a distinct disease entity: ovarian germ cell tumor/mastocytosis with KIT mutations. This report emphasizes the importance of early genetic profiling and multidisciplinary collaboration in diagnosing and managing rare, genetically unified malignancies in pediatric oncology.
    Journal
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    TP53 (Tumor protein P53) • NRAS (Neuroblastoma RAS viral oncogene homolog) • KIT (KIT proto-oncogene, receptor tyrosine kinase) • AFP (Alpha-fetoprotein)
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    TP53 mutation • NRAS mutation • KIT mutation • AFP elevation • NRAS G12 • TP53 Y220C
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    Jakafi (ruxolitinib) • Ayvakit (avapritinib)