Ayvakit (avapritinib)
An approved treatment for Systemic Mastocytosis, Mastocytosis and Gastrointestinal Stromal Tumor.
The same compound appears under different names depending on the context. Here is how to identify Avapritinib wherever you encounter it, plus the key facts at a glance.
- Generic name
- Avapritinib
- Brand name
- Ayvakit
- Development code
- BLU-285
- Drug class
- Kinase inhibitor (KIT and PDGFRA inhibitor)
- Manufacturer
- Blueprint Medicines, a Sanofi company
- How it's taken
- A tablet taken once a day on an empty stomach, at least 1 hour before or 2 hours after a meal.
A once-daily pill for adults with systemic mastocytosis: 25 mg a day for the indolent form (approved May 22, 2023) and 200 mg a day for advanced forms (approved June 16, 2021). It is also approved at 300 mg a day for gastrointestinal stromal tumors (GIST) with a PDGFRA exon 18 mutation, its first approval on January 9, 2020. It is not recommended for people whose platelet count is below 50,000.
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Where Avapritinib fits
For indolent systemic mastocytosis, a once-daily targeted pill added to symptom medicines when moderate to severe symptoms continue. For advanced systemic mastocytosis, one of 2 approved targeted treatments, with midostaurin (Rydapt).
How Avapritinib works
In most adults with systemic mastocytosis, mast cells carry a change in the KIT gene called D816V that keeps the KIT signal switched on, so the cells keep growing and building up. Avapritinib blocks KIT D816V; in lab tests it blocked the mutant form at a concentration about 48 times lower than normal KIT. It also blocks mutant PDGFRA, the driver of some GISTs[1].
Mechanism: Oral kinase inhibitor that blocks KIT D816V, the mutation behind most adult systemic mastocytosis, and mutant PDGFRA in gastrointestinal stromal tumors
Side effects and safety
- Bleeding in the brain. Occurred in 2.9% of people with GIST or advanced systemic mastocytosis and was fatal in under 1%; none of 246 people with indolent disease had one. Get care right away for a severe headache, vomiting, vision changes or confusion. In advanced disease, platelets are checked before starting and every 2 weeks for the first 8 weeks.
- Memory and thinking problems. Forgetfulness, confusion or trouble thinking occurred in 7.8% of people with indolent disease (7% on placebo) and 28% with advanced disease. The dose can be paused or lowered.
- Sensitivity to sunlight. Limit direct sunlight and other UV light during treatment and for 1 week after stopping, and use sunscreen or covering clothes outdoors.
- Harm to an unborn baby. A pregnancy test is done before starting. Use effective birth control during treatment and for 6 weeks after the last dose.
- Platelet count before starting treatment for advanced disease, every 2 weeks for the first 8 weeks, then based on the count
- Watch for headache, vision changes, confusion or memory problems
- Pregnancy test before starting for anyone who can become pregnant
Ayvakit has no boxed warning and no contraindications. The label warns about bleeding in the brain (intracranial hemorrhage), which occurred in 2.9% of 749 people with GIST or advanced systemic mastocytosis and was fatal in under 1%; none of the 246 people with indolent systemic mastocytosis in the PIONEER study had one. It also warns about cognitive effects such as memory problems or confusion (7.8% on Ayvakit against 7% on placebo in indolent disease, 28% in advanced disease), sensitivity to sunlight and possible harm to an unborn baby. In PIONEER, the most common side effects against placebo were swelling around the eyes (13% against 7%), dizziness (13% against 10%), swelling of the arms or legs (12% against 6%) and flushing (11% against 4%)[1].
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Avapritinib
A tablet taken once a day on an empty stomach, at least 1 hour before or 2 hours after a meal. The dose depends on the condition: 25 mg for indolent systemic mastocytosis, 200 mg for advanced systemic mastocytosis and 300 mg for PDGFRA exon 18 GIST. Strong or moderate CYP3A inhibitors and inducers should be avoided, and people taking estrogen birth control may need a low-dose form. Lower doses are used for severe liver impairment[1].
Availability and cost
Only available as the brand-name product.
Help paying for Ayvakit
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Copay help
YourBlueprint Co-Pay Assistance Program: eligible patients with commercial insurance may pay as little as $0 a month; it covers copays, coinsurance and deductibles. Terms and conditions apply.
For: private insurance · source - Free medicine program
Patient Assistance Program: Ayvakit at no cost for eligible people who have no insurance, limited insurance or unaffordable out-of-pocket costs, live in the US or a US territory, have a valid prescription and meet financial criteria.
For: no insurance, underinsured · source - Bridge or quick-start supply
Temporary treatment help: a no-cost supply may be available to eligible patients during a delay or gap in coverage.
For: private insurance, Medicare, Medicaid, TRICARE, no insurance, underinsured · source - Insurance and case manager help
YourBlueprint offers one-on-one support with insurance and financial assistance. Call 1-888-258-7768.
For: private insurance, Medicare, Medicaid, TRICARE, no insurance, underinsured · source
Good to know: Blueprint says Ayvakit is covered by 98% of commercial plans and 99% of Medicare plans, as of February 2026. Blueprint Medicines is now a Sanofi company.
- From a charity · Blood Cancer United (formerly The Leukemia & Lymphoma Society)Acute Myeloid Leukemia Co-Pay Fund (covered diagnoses include Mastocytosis) fundOpen
Pays for: Insurance premiums and treatment-related copays, deductibles and coinsurance, up to $4,000 per year. Requires health insurance (any kind).
Access and eligibility
Approved for adults only; safety and effectiveness in children have not been established. Not recommended for people with a platelet count below 50,000. For GIST, the tumor must carry a PDGFRA exon 18 mutation.
Source: YourBlueprint support for Ayvakit
Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.
Clinical trial results
PIONEER (NCT03731260) compared Ayvakit 25 mg (141 adults) with placebo (71 adults), both on top of usual symptom medicines, for 24 weeks in indolent systemic mastocytosis whose moderate to severe symptoms continued despite at least 2 symptom medicines. The total symptom score, out of 110, fell 15.33 points on Ayvakit against 9.64 on placebo, a difference of 5.69 points. Symptoms fell by half or more in 25% against 10%, and serum tryptase fell by half or more in 53.9% against 0%[1]. In advanced systemic mastocytosis, EXPLORER (NCT02561988) and PATHFINDER (NCT03580655) had no placebo group; of 53 evaluable patients at up to 200 mg a day, 57% responded, and the median response lasted 38.3 months[1].
Development history
Blueprint Medicines developed avapritinib under the code BLU-285. The FDA approved Ayvakit on January 9, 2020 for PDGFRA exon 18 GIST, added advanced systemic mastocytosis on June 16, 2021 and added indolent systemic mastocytosis on May 22, 2023[2]. Blueprint Medicines is now part of Sanofi, and the Ayvakit website describes it as a Sanofi company[3].
Explore Systemic Mastocytosis and Mastocytosis and Gastrointestinal Stromal Tumor trials
Other Systemic Mastocytosis & Mastocytosis treatments
Symptom medicines such as H1 and H2 antihistamines, leukotriene blockers and cromolyn are the base of care, and most PIONEER participants took several. Ayvakit is approved for both indolent and advanced systemic mastocytosis, and Rydapt (midostaurin) for advanced forms. Cogent's bezuclastinib has an FDA decision due December 30, 2026 for nonadvanced systemic mastocytosis.
Common questions about Avapritinib
▸What is Avapritinib (Ayvakit)?
A once-daily pill for adults with systemic mastocytosis: 25 mg a day for the indolent form (approved May 22, 2023) and 200 mg a day for advanced forms (approved June 16, 2021). It is also approved at 300 mg a day for gastrointestinal stromal tumors (GIST) with a PDGFRA exon 18 mutation, its first approval on January 9, 2020. It is not recommended for people whose platelet count is below 50,000.
▸How does Avapritinib work?
In most adults with systemic mastocytosis, mast cells carry a change in the KIT gene called D816V that keeps the KIT signal switched on, so the cells keep growing and building up. Avapritinib blocks KIT D816V; in lab tests it blocked the mutant form at a concentration about 48 times lower than normal KIT. It also blocks mutant PDGFRA, the driver of some GISTs[1].
▸What are the side effects of Avapritinib?
Ayvakit has no boxed warning and no contraindications. The label warns about bleeding in the brain (intracranial hemorrhage), which occurred in 2.9% of 749 people with GIST or advanced systemic mastocytosis and was fatal in under 1%; none of the 246 people with indolent systemic mastocytosis in the PIONEER study had one. It also warns about cognitive effects such as memory problems or confusion (7.8% on Ayvakit against 7% on placebo in indolent disease, 28% in advanced disease), sensitivity to sunlight and possible harm to an unborn baby. In PIONEER, the most common side effects against placebo were swelling around the eyes (13% against 7%), dizziness (13% against 10%), swelling of the arms or legs (12% against 6%) and flushing (11% against 4%)[1].
▸How is Avapritinib taken?
A tablet taken once a day on an empty stomach, at least 1 hour before or 2 hours after a meal. The dose depends on the condition: 25 mg for indolent systemic mastocytosis, 200 mg for advanced systemic mastocytosis and 300 mg for PDGFRA exon 18 GIST. Strong or moderate CYP3A inhibitors and inducers should be avoided, and people taking estrogen birth control may need a low-dose form. Lower doses are used for severe liver impairment[1].
▸Is Avapritinib FDA approved?
Yes, Avapritinib (Ayvakit) is FDA approved (2020) for the treatment of Systemic Mastocytosis and Mastocytosis and Gastrointestinal Stromal Tumor.
▸What dose of Ayvakit is used for indolent systemic mastocytosis?
25 mg once a day, taken on an empty stomach at least 1 hour before or 2 hours after a meal. Advanced systemic mastocytosis uses 200 mg a day, and GIST uses 300 mg a day.
▸Does Ayvakit cause memory problems?
It can. In the PIONEER study of indolent systemic mastocytosis, memory or thinking side effects occurred in 7.8% of people on Ayvakit and 7% on placebo, while in advanced disease at higher doses they occurred in 28%. Tell your doctor about new forgetfulness or confusion, since the dose can be paused or lowered.
▸How is Ayvakit different from Rydapt?
Ayvakit is approved for both indolent and advanced systemic mastocytosis and is taken once a day on an empty stomach. Rydapt (midostaurin) is approved only for advanced forms and is taken twice a day with food. The 2 have not been compared in a head-to-head trial.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- U.S. National Library of Medicine, DailyMed · 2026-08. AYVAKIT (avapritinib) tablets: prescribing information. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=645c887c-8cd4-4623-8da9-ac223d71a8b9
- U.S. Food and Drug Administration. Ayvakit approval history. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=212608
- Blueprint Medicines. YourBlueprint patient support program. https://ayvakit.com/indolent-systemic-mastocytosis/patient-support-program/
- ClinicalTrials.gov. PIONEER: avapritinib versus placebo in indolent systemic mastocytosis. https://clinicaltrials.gov/study/NCT03731260