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Indolent SMAdvanced SMPDGFRA GISTResearch and ResourcesContact a National Account Executive

Get to know the AYVAKIT data
across disease states

Access presentations and publications to stay informed on the evolving research for ISM, AdvSM, PDGFRA GIST, and AYVAKIT data

View the latest AYVAKIT efficacy and safety information

ISM
Efficacy results of AYVAKIT in the PIONEER clinical trial
ISM
Safety data of AYVAKIT in the PIONEER clinical trial
ISM
Evaluation of survival among patients with indolent systemic mastocytosis: a population-level retrospective cohort analysis using healthcare claims dataset
ISM
Impact of avapritinib on polypharmacy for patients with ISM
ISM
Impact of avapritinib on symptom burden in patients with ISM; results from PIONEER
ISM
Impact of avapritinib on biomarkers in ISM; results from PIONEER
ISM
Healthcare resource utilization and costs of Medicare fee-for-service beneficiaries newly diagnosed with moderate to severe ISM
AdvSM
Efficacy results of AYVAKIT in EXPLORER and PATHFINDER clinical trials
AdvSM
Safety results of AYVAKIT in EXPLORER and PATHFINDER clinical trials
AdvSM
Healthcare resource utilization and costs of advanced systemic mastocytosis among Medicare fee-for-service beneficiaries
AdvSM
Duration of treatment and impact on serum tryptase levels in patients with AdvSM treated with avapritinib versus best available therapy
AdvSM
Overall survival in patients with advanced systemic mastocytosis receiving avapritinib versus midostaurin or cladribine
ISMAdvSM
Disease progression in patients with systemic mastocytosis: a US population-level analysis using health claims-based dataset
ISMAdvSM
Systemic mastocytosis: shedding light on a rare and complicated disease
PDGFRA GIST
Avapritinib in unresectable or metastatic PDGFRA D842V-mutant gastrointestinal stromal tumours: long-term efficacy and safety data from the NAVIGATOR Phase I trial

Additional resources

Sample prior authorization policy
Download a sample prior authorization (PA) policy for AYVAKIT
DOWNLOAD SAMPLE PA POLICYDownload resource
NCCN flashcard for AYVAKIT
Download an overview of NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) recommendations for AYVAKIT for the treatment of ISM, AdvSM, and GIST
DOWNLOAD NCCN FLASHCARDDownload resource
AYVAKIT ISM leave behind
Download this resource for ISM disease burden and AYVAKIT information at a glance
DOWNLOAD ISM LEAVE BEHINDDownload resource
AYVAKIT AdvSM leave behind
Download this resource for AdvSM disease burden and AYVAKIT information at a glance
DOWNLOAD AdvSM LEAVE BEHINDDownload resource
Interested in learning more about AYVAKIT? Contact a National Account Executive.

Indication

AYVAKIT® (avapritinib) is indicated for the treatment of adult patients with:

  • Unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations, and
  • Advanced SM (AdvSM) including patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL).
    Limitations of Use: AYVAKIT is not recommended for the treatment of patients with AdvSM with platelet counts of <50 x 109/L.
  • Indolent systemic mastocytosis (ISM).
    Limitations of Use: AYVAKIT is not recommended for the treatment of patients with ISM with platelet counts of <50 x 109/L.

INDICATION & IMPORTANT SAFETY INFORMATION

IMPORTANT SAFETY INFORMATION

Intracranial Hemorrhage — Serious intracranial hemorrhage (ICH) may occur with AYVAKIT treatment; fatal events occurred in <1% of patients. Overall, ICH (e.g., subdural hematoma, ICH, and cerebral hemorrhage) occurred in 2.9% of 749 patients who received AYVAKIT in clinical trials. In GIST patients, ICH occurred in 3 of 267 patients (1.1%) and two (0.7%) of the events were Grade ≥3 and resulted in discontinuation. In AdvSM patients who received AYVAKIT at 200 mg daily, ICH occurred in 2 of 75 patients (2.7%) who had platelet counts ≥50 x 109/L prior to initiation of therapy and in 3 of 80 patients (3.8%) regardless of platelet counts. In ISM patients, no events of ICH occurred in the 246 patients who received any dose of AYVAKIT in the PIONEER study.

Monitor patients closely for risk factors of ICH which may include history of vascular aneurysm, ICH or cerebrovascular accident within the prior year, concomitant use of anticoagulant drugs, or thrombocytopenia. Symptoms of ICH may include headache, nausea, vomiting, vision changes, or altered mental status. Advise patients to seek immediate medical attention for signs or symptoms of ICH. Permanently discontinue AYVAKIT if ICH of any grade occurs.

In AdvSM patients, a platelet count must be performed prior to initiating therapy. AYVAKIT is not recommended in AdvSM patients with platelet counts <50 x 109/L. Following treatment initiation, platelet counts must be performed every 2 weeks for the first 8 weeks. After 8 weeks of treatment, monitor platelet counts every 2 weeks or as clinically indicated based on platelet counts. Manage platelet counts of <50 x 109/L by treatment interruption or dose reduction.

Cognitive Effects — Cognitive adverse reactions can occur in patients receiving AYVAKIT and occurred in 33% of 995 patients overall in patients who received AYVAKIT in clinical trials including: 41% of 601 GIST patients (5% were Grade ≥3), 28% of 148 AdvSM patients (3% were Grade ≥3), and 7.8% of patients with ISM who received AYVAKIT + best supportive care (BSC) versus 7.0% of patients who received placebo + BSC (<1% were Grade 3). Depending on the severity and indication, withhold AYVAKIT and then resume at same dose or at a reduced dose upon improvement, or permanently discontinue.

Photosensitivity — AYVAKIT may cause photosensitivity reactions. In all patients treated with AYVAKIT in clinical trials (n=1049), photosensitivity reactions occurred in 2.5% of patients. Advise patients to limit direct ultraviolet exposure during treatment with AYVAKIT and for one week after discontinuation of treatment.

Embryo-Fetal Toxicity — AYVAKIT can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females and males of reproductive potential to use an effective contraception during treatment with AYVAKIT and for 6 weeks after the final dose of AYVAKIT. Advise women not to breastfeed during treatment with AYVAKIT and for 2 weeks following the final dose.

Adverse Reactions — The most common adverse reactions (≥20%) in patients with unresectable or metastatic GIST were edema, nausea, fatigue/asthenia, cognitive impairment, vomiting, decreased appetite, diarrhea, increased lacrimation, abdominal pain, constipation, rash, dizziness, and hair color changes.

The most common adverse reactions (≥20%) in patients with AdvSM were edema, diarrhea, nausea, and fatigue/asthenia.

The most common adverse reactions (≥10%) in patients with ISM were eye edema, dizziness, peripheral edema, and flushing.

Drug Interactions — Avoid coadministration of AYVAKIT with strong or moderate CYP3A inhibitors. If coadministration with a moderate CYP3A inhibitor cannot be avoided in patients with GIST or AdvSM, reduce dose of AYVAKIT. Avoid coadministration of AYVAKIT with strong or moderate CYP3A inducers. If contraception requires estrogen, limit ethinyl estradiol to ≤20 mcg unless a higher dose is necessary.

To report suspected adverse reactions, contact Blueprint Medicines Corporation at 1-888-258-7768 or the FDA at 1-800-FDA‑1088 or www.fda.gov/medwatch.

AYVAKIT is available in 25-mg, 50-mg, 100-mg, 200-mg and 300-mg tablets.

Please click here to see the full Prescribing Information for AYVAKIT.

Indication

AYVAKIT® (avapritinib) is indicated for the treatment of adult patients with:

  • Unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations, and
  • Advanced SM (AdvSM) including patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL).
    Limitations of Use: AYVAKIT is not recommended for the treatment of patients with AdvSM with platelet counts of <50 x 109/L.
  • Indolent systemic mastocytosis (ISM).
    Limitations of Use: AYVAKIT is not recommended for the treatment of patients with ISM with platelet counts of <50 x 109/L.

INDICATION & IMPORTANT SAFETY INFORMATION

IMPORTANT SAFETY INFORMATION

Intracranial Hemorrhage — Serious intracranial hemorrhage (ICH) may occur with AYVAKIT treatment; fatal events occurred in <1% of patients. Overall, ICH (e.g., subdural hematoma, ICH, and cerebral hemorrhage) occurred in 2.9% of 749 patients who received AYVAKIT in clinical trials. In GIST patients, ICH occurred in 3 of 267 patients (1.1%) and two (0.7%) of the events were Grade ≥3 and resulted in discontinuation. In AdvSM patients who received AYVAKIT at 200 mg daily, ICH occurred in 2 of 75 patients (2.7%) who had platelet counts ≥50 x 109/L prior to initiation of therapy and in 3 of 80 patients (3.8%) regardless of platelet counts. In ISM patients, no events of ICH occurred in the 246 patients who received any dose of AYVAKIT in the PIONEER study.

Monitor patients closely for risk factors of ICH which may include history of vascular aneurysm, ICH or cerebrovascular accident within the prior year, concomitant use of anticoagulant drugs, or thrombocytopenia. Symptoms of ICH may include headache, nausea, vomiting, vision changes, or altered mental status. Advise patients to seek immediate medical attention for signs or symptoms of ICH. Permanently discontinue AYVAKIT if ICH of any grade occurs.

In AdvSM patients, a platelet count must be performed prior to initiating therapy. AYVAKIT is not recommended in AdvSM patients with platelet counts <50 x 109/L. Following treatment initiation, platelet counts must be performed every 2 weeks for the first 8 weeks. After 8 weeks of treatment, monitor platelet counts every 2 weeks or as clinically indicated based on platelet counts. Manage platelet counts of <50 x 109/L by treatment interruption or dose reduction.

Cognitive Effects — Cognitive adverse reactions can occur in patients receiving AYVAKIT and occurred in 33% of 995 patients overall in patients who received AYVAKIT in clinical trials including: 41% of 601 GIST patients (5% were Grade ≥3), 28% of 148 AdvSM patients (3% were Grade ≥3), and 7.8% of patients with ISM who received AYVAKIT + best supportive care (BSC) versus 7.0% of patients who received placebo + BSC (<1% were Grade 3). Depending on the severity and indication, withhold AYVAKIT and then resume at same dose or at a reduced dose upon improvement, or permanently discontinue.

Photosensitivity — AYVAKIT may cause photosensitivity reactions. In all patients treated with AYVAKIT in clinical trials (n=1049), photosensitivity reactions occurred in 2.5% of patients. Advise patients to limit direct ultraviolet exposure during treatment with AYVAKIT and for one week after discontinuation of treatment.

Embryo-Fetal Toxicity — AYVAKIT can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females and males of reproductive potential to use an effective contraception during treatment with AYVAKIT and for 6 weeks after the final dose of AYVAKIT. Advise women not to breastfeed during treatment with AYVAKIT and for 2 weeks following the final dose.

Adverse Reactions — The most common adverse reactions (≥20%) in patients with unresectable or metastatic GIST were edema, nausea, fatigue/asthenia, cognitive impairment, vomiting, decreased appetite, diarrhea, increased lacrimation, abdominal pain, constipation, rash, dizziness, and hair color changes.

The most common adverse reactions (≥20%) in patients with AdvSM were edema, diarrhea, nausea, and fatigue/asthenia.

The most common adverse reactions (≥10%) in patients with ISM were eye edema, dizziness, peripheral edema, and flushing.

Drug Interactions — Avoid coadministration of AYVAKIT with strong or moderate CYP3A inhibitors. If coadministration with a moderate CYP3A inhibitor cannot be avoided in patients with GIST or AdvSM, reduce dose of AYVAKIT. Avoid coadministration of AYVAKIT with strong or moderate CYP3A inducers. If contraception requires estrogen, limit ethinyl estradiol to ≤20 mcg unless a higher dose is necessary.

To report suspected adverse reactions, contact Blueprint Medicines Corporation at 1-888-258-7768 or the FDA at 1-800-FDA‑1088 or www.fda.gov/medwatch.

AYVAKIT is available in 25-mg, 50-mg, 100-mg, 200-mg and 300-mg tablets.

Please click here to see the full Prescribing Information for AYVAKIT.

The information on this website is intended solely for use by US payers, formulary committees, or other similar entities. These materials are not intended to be used by healthcare practitioners for the purpose of making individual patient prescribing or treatment decisions.

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