Ayvakit (avapritinib)
Blueprint Medicines · KIT / PDGFRA Kinase Inhibitor
What is Ayvakit? Ayvakit (avapritinib) is a KIT and PDGFRA kinase inhibitor by Blueprint Medicines. It is FDA-approved for unresectable or metastatic GIST with a PDGFRA exon 18 mutation — including the D842V mutation — and for indolent systemic mastocytosis (ISM).
About Ayvakit
| Brand Name | Ayvakit |
| Generic / INN | avapritinib |
| Drug Class | KIT / PDGFRA Kinase Inhibitor |
| Manufacturer | Blueprint Medicines |
| Administration | Oral tablet |
| FDA Approval | 2020 |
FDA-Approved Uses for Ayvakit
Indications are based on FDA approval. Your healthcare provider determines appropriate use based on your specific diagnosis, stage, biomarker status, and overall clinical picture.
- Unresectable or metastatic gastrointestinal stromal tumor (GIST) with a PDGFRA exon 18 mutation (including D842V)
- Indolent systemic mastocytosis (ISM) — adults
How Does Ayvakit Work?
Avapritinib is a highly selective, potent inhibitor of KIT and PDGFRA kinases, including the PDGFRA D842V mutation that is resistant to imatinib and other prior-generation kinase inhibitors. By blocking PDGFRA D842V and KIT D816V signaling, avapritinib inhibits mast cell and GIST tumor cell proliferation and survival.
Dosage & Administration
GIST: 300 mg orally once daily on an empty stomach (at least 1 hour before or 2 hours after eating). ISM: 25 mg orally once daily with or without food. Dose reductions apply for toxicity. Avoid strong CYP3A inhibitors/inducers.
Dosing is individualized by the prescribing clinician. Never adjust your dose without consulting your healthcare provider. This information is educational only and does not replace the official prescribing information.
Warnings, Precautions & Side Effects
General Warning
Intracranial hemorrhage — monitor for signs and symptoms; withhold if suspected. Cognitive effects (memory impairment, confusional state). Edema. Embryo-fetal toxicity. Do not take with strong CYP3A inhibitors (increases avapritinib exposure significantly).
- Intracranial hemorrhage
- Cognitive effects / CNS toxicity
- Embryo-fetal toxicity
Common Side Effects
- Edema
- Nausea
- Fatigue
- Cognitive effects (memory impairment)
- Vomiting
- Decreased appetite
- Diarrhea
- Hair color changes
This is not a complete list. Always report new or worsening symptoms to your healthcare provider promptly.
Storage & Handling
Store at room temperature 20°C to 25°C (68°F to 77°F). Store below 30°C.
BiologicHealthPlus provides temperature-monitored cold-chain specialty delivery for medications requiring refrigeration. Never use a medication if its storage conditions have been compromised.
Insurance Coverage, Prior Authorization & Patient Assistance
🛡️ Commercial Insurance
Ayvakit is covered by most major commercial insurance plans when medically necessary and prior authorized. Coverage tier and cost-sharing vary by plan.
✅ Prior Authorization
Prior authorization is required by virtually all insurance plans for Ayvakit. BiologicHealthPlus handles the complete prior authorization process at no cost to patients or providers.
🏥 Medicare Coverage
Medicare Part D covers this oral specialty medication. Medicaid coverage varies by state.
🆓 Patient Assistance
Manufacturer patient assistance programs may be available for eligible uninsured or underinsured patients. Contact BiologicHealthPlus for a confidential eligibility review.
Prior Authorization Patient Assistance Insurance & Savings Medication Costs
Frequently Asked Questions — Ayvakit
Ayvakit (avapritinib) is a KIT/PDGFRA inhibitor by Blueprint Medicines approved for PDGFRA exon 18-mutant GIST (including D842V — a mutation resistant to imatinib) and for indolent systemic mastocytosis.
PDGFRA D842V is a specific mutation in gastrointestinal stromal tumors that makes them resistant to most prior GIST therapies (imatinib, sunitinib, regorafenib). Ayvakit was specifically engineered to overcome this resistance and is the only approved therapy specifically for D842V-mutant GIST.
ISM is a chronic condition where mast cells (driven by KIT D816V mutations) accumulate in tissues, causing symptoms such as skin lesions, gastrointestinal problems, and anaphylaxis risk. Ayvakit at 25 mg daily is approved to reduce mast cell burden and symptoms in ISM.
For GIST (300 mg dose): Ayvakit must be taken on an EMPTY stomach. For ISM (25 mg dose): it can be taken with or without food.
Ayvakit is covered by most commercial insurance plans and Medicare Part D with prior authorization and documentation of the required biomarker (PDGFRA exon 18 mutation for GIST).
Related Medications & Conditions
Related Medications
Gleevec (imatinib) · Sutent (sunitinib) · Stivarga (regorafenib) · Qinlock (ripretinib)
Related Conditions
PDGFRA D842V-mutant GIST · Gastrointestinal stromal tumor (GIST) · Indolent systemic mastocytosis (ISM)