For US Healthcare Professionals Only

Frequently asked questions about AYVAKIT as a treatment option for indolent systemic mastocytosis (ISM)

The following is intended to serve as a guide for answering questions frequently asked by healthcare professionals regarding AYVAKIT and its use. This information is intended for US healthcare professionals only and does not represent medical advice. Healthcare professionals should make all diagnostic and clinical decisions in accordance with their medical judgment and individual patient context.

FREQUENTLY ASKED QUESTIONS

What are the most common ISM symptoms?

  • Patients with ISM can experience a variety of symptoms across multiple organ systems5
  • Common symptoms of ISM include skin lesions, itching, anaphylaxis or severe allergic reactions, diarrhea, and nausea5
  • These are not all of the possible symptoms patients with ISM may experience5
Learn more about the burden of indolent systemic mastocytosis >

What is the mechanism of action for AYVAKIT?

  • Avapritinib is a tyrosine kinase inhibitor designed for the potent and selective inhibition of KIT D816V1,9
  • Patients do not need to be KIT D816V positive to receive treatment with AYVAKIT1
Explore how AYVAKIT works >

Is avapritinib chemotherapy?

Avapritinib is a tyrosine kinase inhibitor approved as an oral therapy for adults with indolent systemic mastocytosis.1 Avapritinib is designed to target and inhibit a specific mutation called KIT D816V that drives abnormal growth and division of mast cells.1,18 Conventional, classical chemotherapy works by destroying rapidly growing and dividing cells.18

Explore the mechanism of action >

How was AYVAKIT studied for the treatment of indolent systemic mastocytosis?

PIONEER was a phase 2, multipart, randomized, placebo-controlled, double-blind trial evaluating the safety and efficacy of AYVAKIT 25 mg (N=141) vs placebo (N=71)—both with concomitant best supportive care over 24 weeks in adult patients with indolent systemic mastocytosis.1,9

See study details and efficacy data >

What are the most common adverse reactions to treatment with AYVAKIT?

The most common adverse reactions (10%) in patients with ISM treated with AYVAKIT were eye edema, dizziness, peripheral edema, and flushing.1
Refer to the full Prescribing Information for AYVAKIT for further overall adverse event information.1

Explore the safety data behind AYVAKIT for indolent systemic mastocytosis >

Has intracranial hemorrhage (ICH) been observed with AYVAKIT for indolent systemic mastocytosis?

  • Serious ICH may occur with AYVAKIT treatment; fatal events occurred in <1% of patients1
  • No events of ICH occurred in the 246 patients with ISM who received any dose of AYVAKIT in the PIONEER study1
  • Monitor your patients closely for risk factors of ICH which may include history of vascular aneurysm, ICH or cerebrovascular accident within the prior year, concomitant use of anticoagulant drugs, or thrombocytopenia1
  • Symptoms of ICH may include headache, nausea, vomiting, vision changes, or altered mental status1
  • Advise patients to seek immediate medical attention for signs and symptoms of ICH1
  • Permanently discontinue AYVAKIT if ICH of any grade occurs1

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

Does AYVAKIT affect fertility or the ability to have children?

  • Based on findings from animal studies, AYVAKIT may impair female and male fertility. These findings were not reversible within a 2-month recovery period1
  • Advise females of reproductive potential that AYVAKIT may impair fertility at 200 mg or 300 mg1
  • Advise males of reproductive potential that AYVAKIT may decrease sperm production at 200 mg or 300 mg1

To learn about taking AYVAKIT while pregnant or breastfeeding, please see the full Prescribing Information.

How is AYVAKIT taken?

  • The recommended dosage of AYVAKIT is 25 mg orally, once daily in patients with indolent systemic mastocytosis1
  • Administer AYVAKIT orally on an empty stomach, at least 1 hour before or at least 2 hours after a meal1
  • A modified starting dosage of AYVAKIT is recommended for patients with severe hepatic impairment (Child-Pugh Class C): 25 mg orally every other day1
  • Avoid concomitant use of AYVAKIT with strong or moderate CYP3A inhibitors or inducers1

Learn about the dosing and administration of AYVAKIT and please see the full Prescribing Information.

Does a patient need to stay on AYVAKIT for life?

Indolent systemic mastocytosis is a chronic disease and, as such, AYVAKIT is intended as a chronic treatment.1,7 The Prescribing Information for AYVAKIT does not define a duration of use in indolent systemic mastocytosis.1

What are the AYVAKIT prior authorization requirements?

Approval processes and AYVAKIT prior authorization requirements vary by payer. YourBlueprint® can help navigate the prior authorization process for your patient.

Navigate coverage with YourBlueprint® >

How can YourBlueprint® help my patients?

YourBlueprint® provides dedicated, personalized support to help your eligible patients from day 1 when you enroll them at the time of prescription. Blueprint Medicines offers a series of programs to support patient access including: co-pay support, prior authorization support, coverage interruption, QuickStart, and more.

Access patient support with YourBlueprint® >

What is the SM Provider Peer Directory?

The SM Provider Peer Directory is an online resource that lists the contact information of volunteer healthcare providers who have experience managing patients living with systemic mastocytosis and/or treating patients taking AYVAKIT and are willing to independently connect with peers who have questions about their medical experience.

Have more questions about AYVAKIT for ISM?
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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.

IMPORTANT SAFETY INFORMATION
INDICATION & 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.

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.

References:

  1. AYVAKIT [prescribing information]. Cambridge, MA: Blueprint Medicines Corporation; May 2023.
  2. Kristensen T et al. Am J Hematol. 2014;89(5):493-498.
  3. Garcia-Montero AC et al. Blood. 2006;108(7):2366-2372.
  4. Ungerstedt J et al. Cancers. 2022;14(16):3942.
  5. Pardanani A. Am J Hematol. 2023;98(7):1097-1116.
  6. Data on file. Blueprint Medicines Corporation, Cambridge, MA. 2023.
  7. Gülen T et al. J Intern Med. 2016;279(3):211-228.
  8. Theoharides TC et al. N Engl J Med. 2015;373(2):163-172.
  9. Gotlib J et al. NEJM Evidence. 2023;2(6). Published online May 23, 2023. doi:10.1056/EVIDoa2200339
  10. Gilreath JA et al. Clin Pharmacol. 2019;11:77-92.
  11. Evans EK et al. Sci Transl Med. 2017;9(414):eaao1690.
  12. Padilla B et al. Orphanet J Rare Dis. 2021;16(1):434.
  13. van Anrooij B et al. Allergy. 2016;71(11):1585-1593.
  14. WHO Classification of Tumours Editorial Board. Haematolymphoid tumours [Internet]. Lyon (France): International Agency for Research on Cancer; 2024 [cited April 24, 2024]. (WHO Classification of Tumours Series, 5th ed.; vol. 11). Available from: https://tumourclassification.iarc.who.int/chapters/63
  15. Dranitsaris G et al. J Oncol Pharm Pract. Published online December 27, 2023. doi:10.1177/10781552231221149
  16. Siebenhaar F et al. Immunol Allergy Clin North Am. 2014;34(2):433-447.
  17. Jennings SV et al. Immunol Allergy Clin North Am. 2018;38(3):505-525.
  18. Akin C, ed. Mastocytosis: A Comprehensive Guide. Springer; 2020.