Target the source, not just the symptoms1,2

AYVAKIT is designed to target KIT D816V, the underlying driver of ISM in ~95% of patients1-5

AYVAKIT is a tyrosine kinase inhibitor (TKI) designed for potent and selective inhibition of KIT D816V. AYVAKIT demonstrated greater inhibitory potency for KIT D816V vs wild type KIT in cellular assays.1

Symptom-directed therapies (SDT) alone may not be enough1,2

SDT may help address ISM symptoms, but do not treat the underlying cause of the symptoms, the KIT D816V mutation.

How KIT D816V drives ISM

Mast cells play a key role in the immune system, particularly in allergic responses.6

Stem cell factor (SCF) activates growth, survival, and activation via interaction with the KIT receptor.6

Mast cells degranulate, releasing inflammatory mediators such as leukotriene, histamine, and tryptase.6

SCF activates growth, survival, and activation via interaction with the KIT receptor

In ~95% of ISM cases, the KIT D816V mutation is present, resulting in constant activation, causing an overproduction of abnormal and hyperactive mast cells.2-5

When the KIT D816V mutation is present, it results in constant activation

Abnormal and hyperactive mast cells accumulate throughout the body and release mediators that can lead to potentially debilitating symptoms across organ systems; symptoms such as rash, flushing, brain fog, and diarrhea.2,6,7*

Abnormal and overactive mast cells accumulate and release mediators that can lead to debilitating symptoms

*Not a comprehensive list of symptoms.

How AYVAKIT works

AYVAKIT is a selective TKI inhibitor that is designed to inhibit the constitutive self-activation (autophosphorylation) of the KIT D816V mutation by preventing the overproduction of abnormal and hyperactive mast cells.1

How AYVAKIT inhibits the constructive self-activation of the KIT mutation

AYVAKIT targets the source of the disease thought to cause the symptoms of ISM.1

ISM=indolent systemic mastocytosis; KIT=KIT proto-oncogene, receptor tyrosine kinase.

Consider targeting the cause of the symptoms
when treating ISM1,6,7

See how AYVAKIT was studied in ISM

PIONEER STUDY DESIGN

Patient portrayal

Indolent systemic mastocytosis patient and caregiver

References: 1. AYVAKIT [prescribing information]. Cambridge, MA: Blueprint Medicines Corporation; November 2024. 2. Pardanani A. Am J Hematol. 2023;98(7):1097-1116. 3. Kristensen T et al. Am J Hematol. 2014;89(5):493-498. 4. Garcia-Montero AC et al. Blood. 2006;108(7):2366-2372. 5. Ungerstedt J et al. Cancers. 2022;14(16):3942. 6. Akin C, ed. Mastocytosis: A Comprehensive Guide. Springer; 2020. 7. Gülen T et al. J Intern Med. 2016;279(3):211-228.