Avapritinib

Avapritinib is a 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 (IC 50s ) 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.

• AYVAKIT is a kinase inhibitor indicated for: Gastrointestinal Stromal Tumor (GIST) the treatment of adults with unresectable or metastatic GIST harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations. ( 1.1 , 2.2 ) Advanced Systemic Mastocytosis (AdvSM) the treatment of adult patients with AdvSM.

• None. None.contraindicated

GIST: Select patients for treatment with AYVAKIT based on the presence of a PDGFRA exon 18 mutation. ( 2.2 ) GIST: The recommended dosage is 300 mg orally once daily. ( 2.2 ) AdvSM: The recommended dosage is 200 mg orally once daily. ( 2.3 ) ISM: The recommended dosage is 25 mg orally once daily. ( 2.4 ) Patients with severe hepatic impairment (Child-Pugh Class C): reduce dose of AYVAKIT. ( 2.7 ) 2.1 Recommended Administration Administer AYVAKIT orally on an empty stomach, at least 1 hour before or 2 hours after a meal [see Clinical Pharmacology (12.3) ] . Do not make up for a missed dose within 8 hours of the next scheduled dose. Do not repeat dose if vomiting occurs after AYVAKIT but continue with the next scheduled dose. 2.2 GIST Harboring PDGFRA Exon 18 Mutations Select patients for treatment with AYVAKIT based on the presence of a PDGFRA exon 18 mutation [see Clinical Studies (14.1) ] . An FDA-approved test for the detection of exon 18 mutations is not currently available. The recommended dosage of AYVAKIT is 300 mg orally once daily in patients with GIST . Continue treatment until disease progression or unacceptable toxicity. 2.3 Advanced Systemic Mastocytosis The recommended dosage of AYVAKIT is 200 mg orally once daily in patients with AdvSM. Continue treatment until disease progression or unacceptable toxicity. 2.4 Indolent Systemic Mastocytosis The recommended dosage of AYVAKIT is 25 mg orally once daily in patients with ISM.

Intracranial Hemorrhage : Permanently discontinue for any occurrence of any grade. ( 2.5 , 5.1 ) Cognitive Effects : A broad spectrum of cognitive adverse reactions can occur in patients receiving AYVAKIT. In patients with GIST, AdvSM, or ISM depending on the severity, continue AYVAKIT at same dose, withhold and then resume at same or reduced dose upon improvement, or permanently discontinue. ( 2.5 , 5.2 ) Photosensitivity: May cause photosensitivity reactions. Advise patients to limit direct ultraviolet exposure. ( 5.3 ) Embryo-Fetal Toxicity : Can cause fetal harm. Advise females and males of reproductive potential of the potential risk to a fetus and to use effective contraception. ( 5.4 , 8.1 , 8.3 ) 5.1 Intracranial Hemorrhage Serious intracranial hemorrhage may occur with AYVAKIT treatment; fatal events occurred in less than 1% of patients. Overall, intracranial hemorrhage (e.g., subdural hematoma, intracranial hemorrhage, and cerebral hemorrhage) occurred in 2.9% of the 749 patients with GIST or AdvSM who received AYVAKIT in clinical trials. No events of intracranial hemorrhage occurred in the 246 patients with ISM who received any dose of AYVAKIT in the PIONEER study . Monitor patients closely for risk factors of intracranial hemorrhage which may include history of vascular aneurysm, intracranial hemorrhage or cerebrovascular accident within the prior year, concomitant use of anticoagulant drugs, or thrombocytopenia. Symptoms of intracranial hemorrhage may include headache, nausea, vomiting, vision changes, or altered mental status.

The following clinically significant adverse reactions are described elsewhere in the labeling: Intracranial hemorrhage [see Warnings and Precautions (5.1) ] Cognitive effects [see Warnings and Precautions (5.2) ] Photosensitivity [see Warnings and Precautions (5.3) ] The most common adverse reactions are: GIST (≥20% incidence): edema, nausea, fatigue/asthenia, cognitive impairment, vomiting, decreased appetite, diarrhea, increased lacrimation, abdominal pain, constipation, rash, dizziness, and hair color changes. ( 6.1 ) AdvSM (≥20% incidence): edema, diarrhea, nausea, and fatigue/asthenia. ( 6.1 ) ISM (≥10% incidence): eye edema, dizziness, peripheral edema and flushing. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Blueprint Medicines Corporation at 1-888-258-7768 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The data in the WARNINGS AND PRECAUTIONS reflect exposure to AYVAKIT at 25 mg to 600 mg orally once daily in 995 patients enrolled in one of five clinicals trials conducted in patients with advanced malignancies and systemic mastocytosis, including NAVIGATOR, EXPLORER, PATHFINDER and PIONEER [see Clinical Studies (14.1 , 14.2 , 14.3) ]. These patients included 601 patients with GIST, 148 patients with AdvSM and 246 patients with ISM.

Lactation : Advise not to breastfeed. ( 8.2 ) 8.1 Pregnancy Risk Summary Based on findings from animal studies and its mechanism of action [see Clinical Pharmacology (12.1) ] , AYVAKIT can cause fetal harm when administered to a pregnant woman. There are no available data on AYVAKIT use in pregnant women. Oral administration of avapritinib to pregnant rats during the period of organogenesis was teratogenic and embryotoxic at exposure levels approximately 31.4, 6.3 and 2.7 times the human exposure based on AUC at the 25 mg, 200 mg and 300 mg dose, respectively (see Data ) . Advise pregnant women of the potential risk to a fetus. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively. Data Animal Data In a reproductive toxicity study, administration of avapritinib to rats during the period of organogenesis resulted in decreased fetal body weights, post-implantation loss, and increases in visceral (hydrocephaly, septal defect, and stenosis of the pulmonary trunk) and skeletal (sternum) malformations at doses greater than or equal to 10 mg/kg/day (approximately 31.4, 6.3 and 2.7 times the human exposure based on AUC at the 25 mg, 200 mg and 300 mg dose, respectively).

Metabolism

Avapritinib C max and AUC increased approximately proportionally over the dose range of 25 mg to 400 mg once daily. Steady state concentrations of avapritinib were reached prior to day 15 following daily dosing.

• Jan 9, 2020NDANDA212608Blueprint Medicines

1. 1Fatigue1,65114%
2. 2Off Label Use1,28411%
3. 3Nausea1,16810%
4. 4Diarrhoea9748.5%
5. 5Headache5314.7%
6. 6Dizziness5094.5%
7. 7Swelling Face5084.5%
8. 8Memory Impairment5034.4%
9. 9Vomiting5004.4%
10. 10Asthenia4564.0%
11. 11Peripheral Swelling4473.9%
12. 12Rash4463.9%
13. 13Platelet Count Decreased4433.9%
14. 14Product Dose Omission Issue4353.8%
15. 15Lacrimation Increased4253.7%

Recent PubMed references pinned to Avapritinib as a MeSH major topic. Citations link to pubmed.ncbi.nlm.nih.gov.

• Efficacy and safety of avapritinib in advanced systemic mastocytosis: 4-year follow-up of the PATHFINDER study.
  Blood advances · 2026 · Gotlib J, Reiter A, Radia DH, et al.
  PMID 41604606DOI 10.1182/bloodadvances.2025017519

The 10 most recently updated of 30 ClinicalTrials.gov registrations naming Avapritinib as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Avapritinib for the Treatment of CKIT or PDGFRA Mutation-Positive Locally Advanced or Metastatic Malignant Solid Tumors
  Recruiting · Phase 2 · Interventional · 50 enrolled · M.D. Anderson Cancer Center
  NCT04771520updated 2026-06-11
• A Study Evaluating the Activity of Anti-cancer Treatments Targeting Tumor Molecular Alterations/Characteristics in Advanced / Metastatic Tumors.
  Recruiting · Phase 2 · Interventional · 455 enrolled · Centre Leon Berard
  NCT04116541updated 2026-04-24
• (PATHFINDER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocytosis
  Completed · Phase 2 · Interventional · 107 enrolled · Blueprint Medicines Corporation
  NCT03580655updated 2026-04-24
• An Observational Study in Participants With Indolent Systemic Mastocytosis (ISM)
  Recruiting · Observational · 150 enrolled · Blueprint Medicines Corporation
  NCT07264959updated 2026-04-22
• A Non-Interventional Study in Participants With Indolent Systemic Mastocytosis (ISM) in Germany
  Recruiting · Observational · 80 enrolled · Blueprint Medicines Corporation
  NCT07255638updated 2026-04-20
• Avapritinib With Decitabine in Patients With SM-AHN
  Recruiting · Phase 1 · Interventional · 34 enrolled · H. Lee Moffitt Cancer Center and Research Institute
  NCT06327685updated 2026-04-01
• Safety and Efficacy of Avapritinib in Chinese Patients With Gastrointestinal Stromal Tumor (GIST) in the Real World
  Completed · Observational · 61 enrolled · CStone Pharmaceuticals
  NCT05381753updated 2026-02-11
• Avapritinib Rollover Study
  Recruiting · Phase 4 · Interventional · 60 enrolled · Blueprint Medicines Corporation
  NCT06748001updated 2026-02-09
• A Study of Avapritinib in Pediatric Patients With Solid Tumors Dependent on KIT or PDGFRA Signaling
  Completed · Phase 1 · Phase 2 · Interventional · 29 enrolled · Blueprint Medicines Corporation
  NCT04773782updated 2026-01-13
• (PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo in Patients With Indolent Systemic Mastocytosis
  Active not recruiting · Phase 2 · Interventional · 251 enrolled · Blueprint Medicines Corporation
  NCT03731260updated 2025-09-23

Frequently asked questions

How does Avapritinib work?

Avapritinib is a 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 (IC 50s ) 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.

What is Avapritinib used for?

According to FDA labeling, Avapritinib carries indications including: AYVAKIT is a kinase inhibitor indicated for: Gastrointestinal Stromal Tumor (GIST) the treatment of adults with unresectable or metastatic GIST harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations. ( 1.1 , 2.2 ) Advanced Systemic Mastocytosis (AdvSM) the treatment of adult patients with AdvSM.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Avapritinib?

Avapritinib is classified as Other protein kinase inhibitors, Kinase Inhibitor, Bile Salt Export Pump Inhibitors, Breast Cancer Resistance Protein Inhibitors, Cytochrome P450 2C9 Inhibitors, Multidrug and Toxin Extrusion Transporter 1 Inhibitors, Multidrug and Toxin Extrusion Transporter 2 K Inhibitors, P-Glycoprotein Inhibitors, Platelet-derived Growth Factor alpha Receptor Inhibitors, Tyrosine Kinase Inhibitors, Cellular Proliferation Alteration.

What are the brand names for Avapritinib?

Avapritinib is marketed under brand names including Ayvakit.

What are the contraindications for Avapritinib?

Avapritinib labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.