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Ayvakit (AVAPRITINIB)
Ayvakit works by blocking the activity of the PDGFRA protein, which is involved in the growth and spread of cancer cells.
Ayvakit (avapritinib) is a small molecule kinase inhibitor developed by Blueprint Medicines, targeting the platelet-derived growth factor receptor alpha (PDGFRA). It is a treatment for gastrointestinal stromal tumor (GIST) and systemic mast cell disease, FDA-approved in 2020. Ayvakit is a patented medication with no generic manufacturers available. Its half-life is approximately 32 hours. Key safety considerations include potential side effects such as hypertension, fatigue, and edema.
At a glance
| Generic name | AVAPRITINIB |
|---|---|
| Sponsor | Blueprint Medicines |
| Drug class | Kinase Inhibitor [EPC] |
| Target | Platelet-derived growth factor receptor alpha |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
| First approval | 2020 |
Mechanism of action
Avapritinib is tyrosine kinase inhibitor that targets KIT D816V, PDGFRA and PDGFRA D842 mutants as well as multiple KIT exon 11, 11/17 and 17 mutants with half maximal inhibitory concentrations (IC50s) less than 25 nM in biochemical assays. Certain mutations in PDGFRA and KIT can result in the autophosphorylation and constitutive activation of these receptors which can contribute to tumor and mast cell proliferation. Other potential targets for avapritinib include wild type KIT, PDGFRB, and CSFR1.In cellular assays, avapritinib inhibited the autophosphorylation of KIT D816V with an IC50 of nM, approximately 48-fold lower concentration than wild-type KIT. In cellular assays, avapritinib inhibited the proliferation in KIT mutant cell lines, including murine mastocytoma cell line and human mast cell leukemia cell line. Avapritinib also showed growth inhibitory activity in xenograft model of murine mastocytoma with KIT exon 17 mutation.Avapritinib inhibited the au
Approved indications
- Gastrointestinal stromal tumor
- Systemic mast cell disease
Common side effects
- Edema
- Fatigue/asthenia
- Nausea
- Vomiting
- Diarrhea
- Abdominal pain
- Constipation
- Cognitive impairment
- Dizziness
- Decreased appetite
- Hair color changes
- Increased lacrimation
Key clinical trials
- Avapritinib for the Treatment of CKIT or PDGFRA Mutation-Positive Locally Advanced or Metastatic Malignant Solid Tumors (PHASE2)
- Safety and Efficacy of Avapritinib in Chinese Patients With Gastrointestinal Stromal Tumor (GIST) in the Real World
- A Non-Interventional Study in Participants With Indolent Systemic Mastocytosis (ISM) in Germany
- Avapritinib Rollover Study (PHASE4)
- A Study of Avapritinib in Pediatric Patients With Solid Tumors Dependent on KIT or PDGFRA Signaling (PHASE1,PHASE2)
- Avapritinib With Decitabine in Patients With SM-AHN (PHASE1)
- An Observational Study in Participants With Indolent Systemic Mastocytosis (ISM)
- A Study Evaluating the Activity of Anti-cancer Treatments Targeting Tumor Molecular Alterations/Characteristics in Advanced / Metastatic Tumors. (PHASE2)
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Ayvakit CI brief — competitive landscape report
- Ayvakit updates RSS · CI watch RSS
- Blueprint Medicines portfolio CI