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Indolent systemic mastocytosis (ISM)

Get to know AYVAKIT for your ISM population

The FIRST and ONLY therapy approved for the treatment of adults with ISM

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ISM overview

Systemic mastocytosis (SM) is a rare clonal mast cell neoplasm that may lead to proliferation and activation of abnormal mast cells and is estimated to affect ~32K adults in the United States1-4
Pie graph showing approximately 95 percent
SM is driven by the KIT D816V mutation in ~95% of cases5-7
Pie graph showing approximately 75 percent to 90 percent
ISM is a subtype of SM that represents ~75% to 90% of SM cases4,8-10
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Neurocognitive

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Cardiovascular

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Respiratory

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Gastrointestinal

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Musculoskeletal

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Skin

Your population may experience a wide range of potentially severe and unpredictable ISM symptoms occurring across multiple organ systems1,2,11-13

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AYVAKIT for ISM

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Significant reduction in ISM
symptoms at 6 months14
AYVAKIT + BSC (N=141) demonstrated significant reduction in ISM-SAF TSS vs placebo + BSC (N=71) at Week 24:
  • AYVAKIT + BSC: -15.33 (-18.36, -12.31)
  • Placebo + BSC: -9.64 (-13.61, -5.68)
2-sided P=0.012
Icon of mast cell
Reduction in mast cell burden14
Significantly more patients treated with AYVAKIT + BSC achieved ≥50% reductions in objective measures of mast cell burden at Week 24 vs placebo + BSC:
Serum Tryptase
  • AYVAKIT + BSC (N=141): 53.9%
  • Placebo + BSC (N=71): 0%
KIT D816V VAF*
  • AYVAKIT + BSC (N=118): 67.8%
  • Placebo + BSC (N=63): 6.3%
Bone Marrow Mast Cell Levels†
  • AYVAKIT + BSC (N=106): 52.8%
  • Placebo + BSC (N=57): 22.8%
2-sided P<0.0001 for all endpoints
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Generally well tolerated in a Phase 2 clinical trial14
The most common adverse reactions (>10%) were eye edema, dizziness, peripheral edema, and flushing.
Of all adverse reactions:
  • 55% were Grade 1
  • 38% were Grade 2
  • 7% were Grade 3
  • 0.7% were considered serious
  • 0.7% resulted in permanent discontinuation of AYVAKIT
*Percentage of patients with ≥50% reduction in peripheral blood KIT D816V VAF or undetectable.
†Percentage of patients with ≥50% reduction in BM MCs or no aggregates.
Best supportive care (BSC) is usually referred to as symptom-directed therapies (SDTs) in clinical settings.
PIONEER (N=212) was a Phase 2, multipart, randomized, placebo-controlled, double-blind study evaluating the efficacy and safety of AYVAKIT 25 mg + best supportive care (BSC) (n=141) vs placebo + BSC (n=71) over 24 weeks in adult patients (≥18 years) with a centrally confirmed ISM diagnosis per WHO criteria, and with moderate to severe ISM despite receiving at least 2 symptom-directed therapies (SDTs). Patients were randomized 2:1 to receive once-daily AYVAKIT 25 mg + BSC or placebo + BSC. Patients who completed the 24-week double-blind portion of the trial had the option to enter an open-label extension (OLE) treatment period for up to 5 years and receive once-daily AYVAKIT 25 mg + BSC.14,15
BM=bone marrow; ISM-SAF TSS=Indolent Systemic Mastocytosis Symptom Assessment Form total symptom score; MC=mast cell; VAF=variant allele fraction.
Want to know more? Contact a National Account Executive for more information about AYVAKIT for ISM.
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Learn more about the value of AYVAKIT for ISM

PIONEER clinical trial overview
See AYVAKIT efficacy in the Phase 2 clinical trial
VIEW EFFICACY RESULTSGo to external site
PIONEER clinical trial safety data
Explore the safety of AYVAKIT for ISM
VIEW AYVAKIT SAFETYGo to external site
AYVAKIT ISM leave behind
Download this resource for ISM disease burden and AYVAKIT information at a glance
DOWNLOAD ISM LEAVE BEHINDDownload resource
Presentations and publications from Blueprint Medicines
Access scientific presentation and publication highlights about AYVAKIT for ISM

Indication

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

Cognitive Effects — Cognitive adverse reactions can occur in patients receiving AYVAKIT and occurred in 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, withhold AYVAKIT and then resume at the same dose, or permanently discontinue AYVAKIT.

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. Advise women not to breastfeed during treatment with AYVAKIT and for 2 weeks following the final dose.

Adverse Reactions — 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 or 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 visit www.fda.gov/medwatch.

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

References:

  1. Pardanani A. Systemic mastocytosis in adults: 2023 update on diagnosis, risk stratification and management. Am J Hematol. 2023;98(7):1097-1116.
  2. Theoharides TC, Valent P, Akin C. Mast cells, mastocytosis, and related disorders. N Engl J Med. 2015;373(2):163-172.
  3. Cohen SS, Skovbo S, Vestergaard H, et al. Epidemiology of systemic mastocytosis in Denmark. Br J Haematol. 2014;166(4):521-528.
  4. Data on file. Blueprint Medicines Corporation, Cambridge, MA. 2022.
  5. Garcia-Montero AC, Jara-Acevedo M, Teodosio C, et al. KIT mutation in mast cells and other bone marrow hematopoietic cell lineages in systemic mast cell disorders: a prospective study of the Spanish Network on Mastocytosis (REMA) in a series of 113 patients. Blood. 2006;108(7):2366-2372.
  6. Kristensen T, Vestergaard H, Bindslev-Jensen C, Møller MB, Broesby-Olsen S; Mastocytosis Centre, Odense University Hospital (MastOUH). Sensitive KIT D816V mutation analysis of blood as a diagnostic test in mastocytosis. Am J Hematol. 2014;89(5):493-498.
  7. Evans EK, Gardino AK, Kim JL, et al. A precision therapy against cancers driven by KIT/PDGFRA mutations. Sci Transl Med. 2017;9(414):eaao1690.
  8. Sperr WR, Kundi M, Alvarez-Twose I, et al. International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study. Lancet Haematol. 2019;6(12):e638-e649.
  9. Desmond DH, Carmichael MG. Systemic mastocytosis: the difficult patient with a rare disease. Case presentation and brief review. Hawaii J Med Public Health. 2018;77(2):27-29.
  10. Indolent systemic mastocytosis. Orphanet. Accessed August 16, 2024. https://www.orpha.net/en/disease/detail/98848?name=indolent%20systemic%20mastocytosis&mode=name
  11. Theoharides TC, Tsilioni I, Ren H. Recent advances in our understanding of mast cell activation - or should it be mast cell mediator disorders? Expert Rev Clin Immunol. 2019;15(6):639-656.
  12. Valent P, Akin C, Gleixner KV, et al. Multidisciplinary challenges in mastocytosis and how to address with personalized medicine approaches. Int J Mol Sci. 2019;20(12):2976.
  13. Rossignol J, Polivka L, Maouche-Chrétien L, et al. Recent advances in the understanding and therapeutic management of mastocytosis. F1000Res. 2019;8:F1000 Faculty Rev-1961.
  14. AYVAKIT [prescribing information]. Cambridge, MA: Blueprint Medicines Corporation; May 2023.
  15. Gotlib J, Castells M, Elberink HO, et al. Avapritinib versus placebo in indolent systemic mastocytosis. NEJM Evid. 2023;2(6):EVIDoa2200339.

Indication

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

Cognitive Effects — Cognitive adverse reactions can occur in patients receiving AYVAKIT and occurred in 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, withhold AYVAKIT and then resume at the same dose, or permanently discontinue AYVAKIT.

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. Advise women not to breastfeed during treatment with AYVAKIT and for 2 weeks following the final dose.

Adverse Reactions — 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 or 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 visit www.fda.gov/medwatch.

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

References:

  1. Pardanani A. Systemic mastocytosis in adults: 2023 update on diagnosis, risk stratification and management. Am J Hematol. 2023;98(7):1097-1116.
  2. Theoharides TC, Valent P, Akin C. Mast cells, mastocytosis, and related disorders. N Engl J Med. 2015;373(2):163-172.
  3. Cohen SS, Skovbo S, Vestergaard H, et al. Epidemiology of systemic mastocytosis in Denmark. Br J Haematol. 2014;166(4):521-528.
  4. Data on file. Blueprint Medicines Corporation, Cambridge, MA. 2022.
  5. Garcia-Montero AC, Jara-Acevedo M, Teodosio C, et al. KIT mutation in mast cells and other bone marrow hematopoietic cell lineages in systemic mast cell disorders: a prospective study of the Spanish Network on Mastocytosis (REMA) in a series of 113 patients. Blood. 2006;108(7):2366-2372.
  6. Kristensen T, Vestergaard H, Bindslev-Jensen C, Møller MB, Broesby-Olsen S; Mastocytosis Centre, Odense University Hospital (MastOUH). Sensitive KIT D816V mutation analysis of blood as a diagnostic test in mastocytosis. Am J Hematol. 2014;89(5):493-498.
  7. Evans EK, Gardino AK, Kim JL, et al. A precision therapy against cancers driven by KIT/PDGFRA mutations. Sci Transl Med. 2017;9(414):eaao1690.
  8. Sperr WR, Kundi M, Alvarez-Twose I, et al. International prognostic scoring system for mastocytosis (IPSM): a retrospective cohort study. Lancet Haematol. 2019;6(12):e638-e649.
  9. Desmond DH, Carmichael MG. Systemic mastocytosis: the difficult patient with a rare disease. Case presentation and brief review. Hawaii J Med Public Health. 2018;77(2):27-29.
  10. Indolent systemic mastocytosis. Orphanet. Accessed August 16, 2024. https://www.orpha.net/en/disease/detail/98848?name=indolent%20systemic%20mastocytosis&mode=name
  11. Theoharides TC, Tsilioni I, Ren H. Recent advances in our understanding of mast cell activation - or should it be mast cell mediator disorders? Expert Rev Clin Immunol. 2019;15(6):639-656.
  12. Valent P, Akin C, Gleixner KV, et al. Multidisciplinary challenges in mastocytosis and how to address with personalized medicine approaches. Int J Mol Sci. 2019;20(12):2976.
  13. Rossignol J, Polivka L, Maouche-Chrétien L, et al. Recent advances in the understanding and therapeutic management of mastocytosis. F1000Res. 2019;8:F1000 Faculty Rev-1961.
  14. AYVAKIT [prescribing information]. Cambridge, MA: Blueprint Medicines Corporation; May 2023.
  15. Gotlib J, Castells M, Elberink HO, et al. Avapritinib versus placebo in indolent systemic mastocytosis. NEJM Evid. 2023;2(6):EVIDoa2200339.

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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