Sign up for updates
  Efficacy Data   
Clinical Trial DesignOverall Response
Rate
  Safety Data  
  Dosing   
Starting
AyvakitPlatelet
MonitoringDose
ModificationsTherapy
Management

  Patient Profiles   
WHO CriteriaPatient Profiles
  Resources   
Video LibraryDownloadable MaterialsYour Blueprint Patient SupportContact a Representative
Indolent ISMAdvanced SMPDGFRA GIST
AYVAKIT® 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 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=National Comprehensive Cancer Network.

57 percent 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 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

Watch video of Dr. Mesa review information about AYVAKIT® efficacy and safety data
Watch Dr Mesa review information about efficacy and safety data.
Watch Now

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

Patients
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
Medicine bottle and tablet
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
Down Arrow
View baseline demographic characteristics (N=53)1
Down Arrow
Efficacy data

Proven efficacy across all subtypes and regardless of prior antineoplastic therapy1,7

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

57 percent overall response rate

(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.

icon triangle down navy
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.

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