FAQs

• Indolent systemic mastocysis is a condition in which an overproduction of mutated and hyperactive mast cells creates disruption throughout the body, which can lead to various debilitating symptoms across multiple organ systems^1-3
• The KIT D816V mutation is typically the culprit in indolent systemic mastocysis, driving overproduction of abnormal mast cells in ~95% of cases^1,4-6

• Patients with indolent systemic mastocytosis can experience a variety of symptoms across multiple organ systems¹
• Common symptoms of indolent systemic mastocytosis include skin lesions, itching, anaphylaxis or severe allergic reactions, diarrhea, and nausea¹
• These are not all of the possible symptoms patients with indolent systemic mastocytosis may experience¹

• Indolent systemic mastocytosis may lead to serious health consequences and symptoms can worsen over time⁷
• In a survey of 40 adult patients with indolent systemic mastocytosis, 55% had a higher frequency of symptoms and 47.5% had greater severity of symptoms since diagnosis⁷*

*Survey data were collected from 40 adults with indolent systemic mastocytosis (ISM) meeting the WHO diagnostic criteria, including the validated ISM-SAF and the 12-item Short-Form Health Survey. ISM burden was analyzed by comparing moderate to severe TSS scores with mild TSS scores using Kruskal-Wallis and Fisher’s exact tests.⁷

• Per the World Health Organization and International Consensus Classification, KIT D816V is one of the minor criteria for diagnosing systemic mastocytosis¹
• KIT D816V testing should be conducted using a high-sensitivity molecular assay⁸
• Despite high-sensitivity assays available, KIT D816V can still be missed due to false-negative or undetectable results⁸
• An undetectable KIT D816V result in the peripheral blood does not rule out SM, as circulating mutated mast cells may be rare and evade detection⁸

• Per the World Health Organization and International Consensus Classification, one of the minor criteria for diagnosing SM is an elevated serum tryptase level >20 ng/mL^1,9
• Despite being a minor criterion for SM diagnosis, a tryptase of <20 ng/mL does not rule out systemic mastocytosis, as tryptase levels are highly variable within the SM population^10-12
• Approximately 30% of patients with indolent systemic mastocytosis exhibit a serum tryptase level <20 ng/mL¹³*

*Survey data were collected from 40 adults with ISM meeting the WHO diagnostic criteria, including the validated ISM-SAF and the 12-item Short-Form Health Survey. ISM burden was analyzed by comparing moderate to severe TSS scores with mild TSS scores using Kruskal-Wallis and Fisher’s exact tests.

• Symptom-directed therapies may help address indolent systemic mastocytosis symptoms, but do not treat the underlying cause of ISM^1,14 
• AYVAKIT is a tyrosine kinase inhibitor (TKI) that is a potent and selective inhibitor of KIT D816V. In cellular assays, AYVAKIT demonstrated greater inhibitory potency for KIT D816V vs wild type KIT, which ultimately blocks overproduction of mutated and hyperactive mast cells¹⁴ 
• AYVAKIT is the only FDA-approved treatment for adults with indolent systemic mastocytosis and can be used regardless of treatment line. In the PIONEER clinical trial, adding AYVAKIT to BSC resulted in significantly greater symptom reduction vs placebo + BSC at 24 weeks¹⁴ 

• At 6 months, patients in the clinical trial who added AYVAKIT to their current BSC saw significantly greater symptom reduction vs placebo + BSC¹⁴

• Yes, patients can take AYVAKIT concomitantly with their current symptom-directed therapies¹⁰
• In PIONEER, patients receiving AYVAKIT or placebo were first optimized on a range of symptom-directed therapies, including¹⁰:
  • Anti-immunoglobulin E antibody (omalizumab)
  • Glucocorticoids
  • Cromolyn sodium
  • H1 antihistamines
  • H2 antihistamines
  • Leukotriene inhibitors
  • Proton pump inhibitors

• No, patients do not need to be positive for the KIT D816V mutation to start AYVAKIT treatment¹⁴
• 93% of patients in the AYVAKIT + BSC arm of the PIONEER clinical trial were KIT D816V positive, with 7% being undetectable¹⁴

• A heterogeneous population of patients living with indolent systemic mastocytosis was reflected in PIONEER, with varied serum tryptase levels at baseline across both trial arms^10,14
• In the AYVAKIT + BSC arm, serum tryptase level ranged from 3.6-256 ng/mL, with a median of 38.4 ng/mL^10,14
• In the placebo + BSC arm, serum tryptase level ranged from 5.7-501.6 ng/mL, with a median of 43.7 ng/mL^10,14
• 20% of patients in PIONEER (n=43/212) had a serum tryptase level <20.0 ng/mL at baseline¹⁰

• The most common adverse reactions (≥10%) in patients with indolent systemic mastocytosis treated with AYVAKIT were eye edema, dizziness, peripheral edema, and flushing¹⁴
• Serious adverse reactions occurred in 1 patient (0.7%) who received AYVAKIT due to pelvic hematoma¹⁴
• Permanent discontinuation of AYVAKIT due to an adverse reaction occurred in 1 patient (0.7%) due to dyspnea and dizziness¹⁴
• Refer to the full Prescribing Information for AYVAKIT for further overall adverse event information on indolent systemic mastocytosis

• No events of ICH have been reported in patients with indolent systemic mastocytosis who received AYVAKIT in the PIONEER study¹⁴

Please see the Important Safety Information and the full Prescribing Information for AYVAKIT for additional information on ICH and other Warnings and Precautions.

• AYVAKIT should not be taken while pregnant.¹⁴ While there is no contraindication to becoming pregnant after stopping AYVAKIT treatment, consider the following for patients currently taking AYVAKIT¹⁴:
  • Based on findings from animal studies and its mechanism of action, AYVAKIT can cause fetal harm when administered to pregnant women.
    Advise pregnant women of the potential risk to a fetus
  • Advise females and males with female partners of reproductive potential to use 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

• In a nonclinical organ assessment study, cysts in the ovary and lower sperm counts were observed in animals after they were given AYVAKIT, which potentially could impact fertility. Following a 2-month recovery period, these observations were not reversible¹⁴
• In a nonclinical animal study to assess fertility, AYVAKIT did not impair the ability to become pregnant¹⁴
• Advise females and males of reproductive potential that AYVAKIT may impair ability to become pregnant after stopping AYVAKIT 200-mg or 300-mg doses. The recommended dose for patients with indolent systemic mastocysis is 25 mg¹⁴
• It is important to remind patients not to become pregnant while taking AYVAKIT or within 6 weeks after stopping AYVAKIT¹⁴

• No, AYVAKIT is not considered a traditional chemotherapy treatment^3,14
• AYVAKIT is in a class of medications called tyrosine kinase inhibitors (TKIs) and is designed to target the KIT D816V mutation—the primary driver of indolent systemic mastocysis^1-3,14,15

• Indolent systemic mastocysis is a chronic disease, and patients should remain on AYVAKIT treatment until their healthcare provider tells them to stop^2,3,14
• The Prescribing Information for AYVAKIT does not define a duration of use in indolent systemic mastocytosis^2,3,14

• AYVAKIT is the first and only FDA-approved therapy for adults with indolent systemic mastocytosis¹⁴ 
• AYVAKIT can be taken in addition to a patient’s symptom-directed therapies¹⁰ 
• AYVAKIT works at the source: It blocks the overproduction of abnormal hyperactive mast cells—the cause of indolent systemic mastocytosis symptoms^4-6,14
• It is important to remind your patients to take AYVAKIT consistently as prescribed: AYVAKIT is intended to be taken orally, once daily, not on an as-needed basis¹⁴ 
• AYVAKIT is experienced over time: It may take time for patients taking AYVAKIT to see results. In the clinical trial, patients taking AYVAKIT + BSC saw a significant reduction in symptom burden vs patients taking placebo + BSC at 6 months¹⁴

• Live and virtual patient education events: Blueprint Medicines sponsors live and virtual educational events designed to help educate patients with indolent systemic mastocytosis about AYVAKIT as a treatment option where they can hear from an HCP and a patient currently taking AYVAKIT. Patients can go to ayvakit.com/indolent-systemic-mastocytosis/events to register and also learn about upcoming events
• AYVAKIT ISM Mentor Program: Patients can connect with another patient who is currently taking AYVAKIT to ask questions and hear about their journey to diagnosis, AYVAKIT experience, and support options. Patients can call 1-833-476-6338 to learn more

• The recommended dosage of AYVAKIT is 25 mg orally, once daily in patients with indolent systemic mastocytosis¹⁴
• Administer AYVAKIT orally on an empty stomach, at least 1 hour before or at least 2 hours after a meal¹⁴
• A modified starting dosage of AYVAKIT is recommended for patients with severe hepatic impairment (Child-Pugh Class C): 25 mg orally every other day¹⁴
• 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 necessary¹⁴

• No, there are no lab monitoring requirements for adult patients with indolent systemic mastocytosis in the prescribing information for AYVAKIT¹⁴
• However, AYVAKIT is not recommended for patients with indolent systemic mastocytosis with platelet counts <50 x 10⁹/L¹⁴ 

Please see the Important Safety Information and the full Prescribing Information for AYVAKIT for additional guidance.

• Yes, YourBlueprint® provides dedicated, personalized support to help your patients from Day 1
• YourBlueprint may be able to help your eligible patients access AYVAKIT. Enroll your patients at time of prescription to support the patient experience through our programs:
  • Co-pay Support
  • Coverage Interruption
  • QuickStart
  • Patient Assistance Program
  • Case Managers can also help your patients through nonclinical aspects of therapy by providing 1:1 support calls and patient education resources

• Yes, there is a co-pay program for your patients. Eligible patients with commercial insurance may be able to reduce their out-of-pocket costs (co-pay, co-insurance, or deductible) to as little as $0 per fill up to an annual maximum of $25,000*
• Over 99% of commercial insurance plans and 99% of Medicare plans cover AYVAKIT^†
• AYVAKIT will require a prior authorization with the enrollment form, which includes:
  • Diagnosis codes
  • Lab results (platelet counts, per label)
  • Clinic notes with SM subtype
  • Most denials for AYVAKIT are due to missing or incomplete information. When working on insurance coverage approval for AYVAKIT, YourBlueprint® and our network of specialty pharmacies can help support your patient through the process of managing a prior authorization requirement 

*Terms and conditions apply.

^†Coverage data as of December 2024.

• The SM Provider Peer Directory is an online resource that lists the contact information of US healthcare providers who have attested that they have experience managing patients living with systemic mastocytosis and/or treating patients taking AYVAKIT
• These providers have volunteered to independently connect with peers who have questions about their medical experience
• This Directory is intended for HCPs only and is not intended as a resource or referral source for patients