Efficacy
demonstrated in
EXPLORER
and PATHFINDER
clinical trials1
NCCN
RECOMMENDED
NCCN
RECOMMENDED
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
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 a 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.
Median Age | 67 years (37-85) |
---|---|
Gender | 58% male, 42% female |
ECOG PS | 0-1: 68% |
2-3: 32% | |
Ongoing corticosteroid use | 40% |
Presence of KIT D816V mutation | 94% (as measured by ddPCR) |
Prior antineoplastic therapy | 66% |
Prior midostaurin | 47% |
Advanced SM subtypes | ASM: 3.8% (n=2) |
SM-AHN: 75.5% (n=40) | |
MCL: 20.7% (n=11) | |
Baseline platelet count ≥50 x 109/L | 91% |
Proven efficacy
across all subtypes
and regardless of prior antineoplastic therapy1,6
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All subtypes: ASM, MCL, SM-AHN (n=53)
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):
2.1 months - Median time to CR and CRh (n=15)6:
9.2 months
a Median duration of follow-up was 11.6 months (95% CI: 9.9 to 16.3 months).
b 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 criteria.7

Aggressive systemic mastocytosis (ASM) (n=2)
- 1/2 patients achieved CR/CRh

Mast cell leukemia (MCL)
(n=11)

Systemic mastocytosis with an associated hematological neoplasm (SM-AHN) (n=40)
CI=confidence interval; CR=complete remission; CRh=complete remission with partial hematologic recovery; 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.
In a pre-planned subgroup analysis, AYVAKIT demonstrated efficacy regardless of antineoplastic therapy.6
- For treatment-naïve 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%)
The safety of AYVAKIT was evaluated in the EXPLORER and PATHFINDER clinical trials.

IMPORTANT SAFETY INFORMATION
There are no contraindications for AYVAKIT.
Serious intracranial hemorrhage (ICH) may occur with AYVAKIT treatment; fatal events occurred in <1% of patients. Overall, ICH (eg, subdural hematoma, ICH, and cerebral hemorrhage) occurred in 2.9% of 749 patients who received AYVAKIT. 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 of ICH including those with thrombocytopenia, vascular aneurysm or a history of ICH or cerebrovascular accident within the prior year. 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 adverse reactions can occur in patients receiving AYVAKIT. Cognitive adverse reactions occurred in 39% of 749 patients and in 28% of 148 SM patients (3% were Grade >3). Memory impairment occurred in 16% of patients; all events were Grade 1 or 2. Cognitive disorder occurred in 10% of patients; <1% of these events were Grade 3. Confusional state occurred in 6% of patients; <1% of these events were Grade 3. Other events occurred in < 2% of patients. Depending on the severity, withhold AYVAKIT and then resume at same dose or at a reduced dose upon improvement, or permanently discontinue.
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 method of 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 after the final dose.
The most common adverse reactions (≥20%) at all doses were edema, diarrhea, nausea and fatigue/asthenia.
Avoid coadministration of AYVAKIT with strong and moderate CYP3A inhibitors. If coadministration with a moderate CYP3A inhibitor cannot be avoided, reduce dose of AYVAKIT. Avoid coadministration of AYVAKIT with strong and moderate CYP3A inducers.
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.
Please click here to see the full Prescribing Information for AYVAKIT.
INDICATION
AYVAKIT 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.
References:
- AYVAKIT [prescribing information]. Cambridge, MA: Blueprint Medicines Corporation; June 2021.
- Gilreath JA, et al. Clin Pharmacol. 2019;11:77-92.
- Garcia-Montero AC, et al. Blood. 2006;108(7):2366-2372.
- Verstovsek S. Eur J Haematol. 2013;90(2):89-98.
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