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Indolent ISMAdvanced SMPDGFRA GIST
NCCN
GUIDELINES®
RECOMMENDED
NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) recommend avapritinib (AYVAKIT) (if platelets ≥50 x 109/L) as an NCCN Category 2A, preferred first-line treatment option for advanced systemic mastocytosis (SM), including aggressive SM (ASM), SM with an associated hematologic neoplasm (SM-AHN) (when the SM component requires prioritization over the AHN component), and mast cell leukemia (MCL).2*

*NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way.

NCCN=National Comprehensive Cancer Network.


57% Overall Response Rate

(95% CI: 42%, 70%)†

Proven efficacy and demonstrated duration of response1

38.3-month median duration of response (DOR) demonstrated in clinical trials (95% CI: 19, NE) DRIVEN PRIMARILY by SM-AHN patients1

EXPLORER and PATHFINDER were 2 multicenter, single-arm, open-label clinical trials evaluating the efficacy and safety of AYVAKIT for patients with Advanced SM.1

Dr. Ruben Mesa of the AYVAKIT® (avapritinib) Expert Series
Watch Dr Mesa review information about efficacy and safety data.
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Efficacy was based on overall response rate (ORR) per modified IWG-MRT-ECNM criteria across all evaluable patients dosed up to 200 mg daily (N=53). In the subgroup of patients with MCL, the efficacy was based on complete remission (CR).1

53 patients were evaluable for a response across the 2 trials, with a median follow-up of 11.6 months (95% CI: 9.9 to 16.3 months)1

Patients enrolled in EXPLORER received a starting dose of AYVAKIT ranging from 30 mg to 400 mg orally once daily. In PATHFINDER, patients were enrolled at the recommended starting dose of 200 mg orally once daily1‡

†ORR per modified IWG-MRT-ECNM is defined as patients who achieved a CR, CRh, or PR.
‡The recommended dosage of AYVAKIT for patients with AdvSM is 200 mg.


View criteria for response-evaluable patients1
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View baseline demographic characteristics (N=53)1
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Proven efficacy across all subtypes and regardless of prior antineoplastic therapy1,3

Proven efficacy and demonstrated duration of response in Advanced SM1

ORR across all evaluable patients with Advanced SM (N=53)§ who were dosed up to 200 mg daily1,3

CR+CRh: 28%

PR: 28%

Clinical improvement: 15%

57% Overall Response Rate

(95% CI: 42%, 70%)

CR+CRh: 28%

PR: 28%

Clinical improvement: 15%

72% ORR was achieved with the addition of patients who had clinical improvement||

ORR per modified IWG-MRT-ECNM is defined as patients who achieved a CR, CRh, or PR. Modified IWG criteria evaluated overall response rate by ≥12 weeks’ response duration, resolution of ≥1 findings of nonhematologic and hematologic organ damage (C-findings), and ≥50% reduction in mast cell burden and serum tryptase (must be <20 ng/mL if baseline was ≥40 ng/mL for CR/CRh).

§Median duration of follow-up was 11.6 months (95% CI: 9.9, 16.3).

∥Clinical improvement is defined as having a response duration of ≥12 weeks and fulfillment of 1 or more of the nonhematologic and/or hematologic response criteria4

Median DOR in
the overall population

38.3

MONTHS

(95% CI: 19, NE)1

Median time to
response

2.1

MONTHS

(n=30)1

Median time to
CR and CRh

9.2

MONTHS

(n=15)3

Proven efficacy across subtypes and regardless of prior antineoplastic therapy1

MCL (n=11)1

45% ORR

(95% CI: 17%, 77%)

SM-AHN (n=40)1

58% ORR

(95% CI: 41%, 73%)

78% ORR was achieved with the
addition of patients who had a
clinical improvement1

ASM (n=2)1

100% ORR

(95% CI: 16%, 100%)

1 of 2 patients
achieved CR/CRh1

In a preplanned subgroup analysis, AYVAKIT demonstrated efficacy regardless of prior antineoplastic therapy3

In treatment-naive patients (n=18), ORR
was 72.2% (95% CI: 46.5%, 90.3%)

In patients with prior antineoplastic therapy
(including midostaurin) (n=35), ORR was
48.6% (95% CI: 31.4%, 66%)

see the PATHFINDER 3-year follow-up

Individual results may vary.



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Learn about the safety of AYVAKIT evaluated in the
EXPLORER and PATHFINDER clinical trials.
Go To Safety Data

References: 1. AYVAKIT [prescribing information]. Cambridge, MA: Blueprint Medicines Corporation; November 2024. 2. Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Systemic Mastocytosis V.1.2025. © National Comprehensive Cancer Network, Inc. 2025. All rights reserved. Accessed February 21, 2025. To view the most recent and complete version of the guideline, go online to NCCN.org. NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way. 3. Data on file. Blueprint Medicines Corporation, Cambridge, MA. 4. Gotlib J et al. Blood. 2013;121(13):‍2393-2401.

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INDICATION

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

IMPORTANT SAFETY INFORMATION
INDICATION & 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 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.

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.

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 28% of 148 AdvSM patients (3% 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%) were edema, diarrhea, nausea, and fatigue/asthenia.

Drug Interactions—Avoid coadministration of AYVAKIT with strong or moderate CYP3A inhibitors. If coadministration with a moderate CYP3A inhibitor cannot be avoided, 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, and 200-mg tablets.

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