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Indolent ISMAdvanced SMPDGFRA GIST
AYVAKIT® (avapritinib) 25 mg white logo

Efficacy demonstrated in EXPLORER and PATHFINDER clinical trials1

NCCN
RECOMMENDED
NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) recommend avapritinib (AYVAKIT) (if platelets ≥50 x 109/L) as a 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 more immediate treatment), and mast cell leukemia (MCL).2

NCCN=National Comprehensive Cancer Network.

57% ORR Icon

(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 patients

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

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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

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53 patients were evaluable for a response, with a median follow-up of 11.6 months (95% CI: 9.9 to 16.3 months)1
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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 for patients with Advanced SM is 200 mg orally once daily per the US Prescribing Information.

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 therapy1,7

All subtypes: ASM, MCL, SM-AHN (N=53)

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(95% CI: 42%, 70%)a

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

Median DOR of 38.3 months (95% CI: 38, NE)

  • Median time to response (n=30)7:
    2.1 months
  • Median time to CR and CRh (n=15)7: 9.2 months

CR+CRh: 28%

PR: 28%

Clinical improvement: 15%

aMedian duration of follow-up was 11.6 months (95% CI: 9.9 to 16.3 months).

bClinical improvement is defined as having a response duration of ≥12 weeks and fulfillment of 1 or more of the nonhematologic and/or hematologic response criteria.8

CI=confidence interval; CR=complete remission; CRh=complete remission with partial hematologic recovery; ddPCR=droplet digital polymerase chain reaction; DOR=duration of response; ECNM=European Competence Network on Mastocytosis; ECOG PS=Eastern Cooperative Oncology Group Performance Status; IWG-MRT=International Working Group-Myeloproliferative Neoplasms Research and Treatment; NE=not estimable; PR=partial remission; WHO=World Health Organization.

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In a pre-planned subgroup analysis, AYVAKIT demonstrated efficacy regardless of antineoplastic therapy.7

  • For treatment-naive patients (n=18), ORR was 72.2% (95% CI: 46.5%, 90.3%)
  • For patients with prior antineoplastic therapy (including midostaurin) (n=35), ORR was 48.6% (95% CI: 31.4%, 66.0%)

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