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Gastrointestinal stromal tumors with PDGFRA mutation (PDGFRA GIST)

Get to know AYVAKIT for your PDGFRA GIST population

The FIRST approved therapy for GIST caused by a PDGFRA exon 18 mutation including the D842V mutation

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About PDGFRA GIST

Icon of intestines

PDGFRA GIST is a rare disease that affects the production of cells in the gastrointestinal tract known as interstitial cells of Cajal (ICC)1

Icon of stomach

With PDGFRA GIST, a genetic mutation makes ICCs grow out of control. This forms a tumor, most often in the stomach or small intestine1

~10%-15%

KIT and PDGFRA are common gene mutations that drive GISTs. PDGFRA mutations occur in ~10% to 15% of GIST cases. One of the most frequent types of PDGFRA mutations occur in exon 18, including the D842V mutation1

While PDGFRA GIST symptoms vary, common symptoms include2-5:
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Blood in vomit
and/or stool

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Abdominal pain
and swelling

Icon of person with difficulty swallowing

Difficulty
swallowing

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Loss of appetite and
weight loss

Historically, patients with unresectable or metastatic D842V-mutant GIST had a poor prognosis6:

Icon of timeline with 3 to 5 months highlighted as median progression-free survival, and 15 months highlighted as the approximate overall survival
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AYVAKIT for PDGFRA GIST

AYVAKIT targets abnormal proteins involved in the growth of GIST that are caused by KIT and PDGFRA gene mutation6
targets
binds
inhibits
AYVAKIT is the only approved PDGFRA exon 18 GIST treatment that binds to these abnormal proteins while they are in their “on” state. It targets, binds to, and inhibits the specific proteins that lead to the growth and spread of GIST cancer cells6,7

Want to know more? Contact a National Account Executive for more information about AYVAKIT for PDGFRA GIST.

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Learn more about the value of AYVAKIT for PDGFRA GIST

NAVIGATOR clinical trial overview
See AYVAKIT efficacy and safety in a Phase 1 clinical study

INDICATION

AYVAKIT® (avapritinib) is indicated for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations.

INDICATION & IMPORTANT SAFETY INFORMATION

IMPORTANT SAFETY INFORMATION

Intracranial Hemorrhage — Serious intracranial hemorrhage (ICH) may occur with AYVAKIT treatment; fatal events occurred in <1% of patients. Overall, ICH (e.g., subdural hematoma, ICH, and cerebral hemorrhage) occurred in 2.9% of 749 patients who received AYVAKIT in clinical trials. In GIST patients, ICH occurred in 3 of 267 patients (1.1%) and two (0.7%) of the events were Grade ≥3 and resulted in discontinuation.

Monitor 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 thrombocytopenia. Symptoms of ICH may include headache, nausea, vomiting, vision changes, or altered mental status. Advise patients to seek immediate medical attention for signs or symptoms of ICH. Permanently discontinue AYVAKIT if ICH of any grade occurs.

Cognitive Effects — Cognitive adverse reactions can occur in patients receiving AYVAKIT and occurred in 33% of 995 patients overall in patients who received AYVAKIT in clinical trials including: 41% of 601 GIST patients (5% were Grade ≥3). Depending on the severity, withhold AYVAKIT and then resume at same dose or at a reduced dose upon improvement, or permanently discontinue.

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 of AYVAKIT. Advise women not to breastfeed during treatment with AYVAKIT and for 2 weeks following the final dose.

Adverse Reactions — The most common adverse reactions (≥20%) were edema, nausea, fatigue/asthenia, cognitive impairment, vomiting, decreased appetite, diarrhea, increased lacrimation, abdominal pain, constipation, rash, dizziness, and hair color changes.

Drug Interactions — Avoid coadministration of AYVAKIT with strong or moderate CYP3A inhibitors. If coadministration with a moderate CYP3A inhibitor cannot be avoided, reduce dose of AYVAKIT. Avoid coadministration of AYVAKIT with strong or moderate CYP3A inducers. If contraception requires estrogen, limit ethinyl estradiol to ≤20 mcg unless a higher dose is necessary.

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.

AYVAKIT is available in 100-mg, 200-mg and 300-mg tablets.

Please click here to see the full Prescribing Information for AYVAKIT.

References:

  1. Sun Y, Yue L, Xu P, Hu W. An overview of agents and treatments for PDGFRA-mutated gastrointestinal stromal tumors. Front Oncol. 2022;12:927587.
  2. Stamatakos M, Douzinas E, Stefanaki C, et al. Gastrointestinal stromal tumor. World J Surg Oncol. 2009;7:61.
  3. Akahoshi K, Oya M, Koga T, Shiratsuchi Y. Current clinical management of gastrointestinal stromal tumor. World J Gastroenterol. 2018;24(26):2806-2817.
  4. American Cancer Society. Gastrointestinal stromal tumor early detection, diagnosis, and staging. Accessed August 16, 2024. https://www.cancer.org/cancer/types/gastrointestinal-stromal-tumor/detection-diagnosis-staging.html
  5. Nishida T, Blay JY, Hirota S, Kitagawa Y, Kang YK. The standard diagnosis, treatment, and follow-up of gastrointestinal stromal tumors based on guidelines. Gastric Cancer. 2016;19(1):3-14.
  6. Heinrich MC, Jones RL, von Mehren M, et al. Avapritinib in advanced PDGFRA D842V-mutant gastrointestinal stromal tumour (NAVIGATOR): a multicentre, open-label, phase 1 trial. Lancet Oncol. 2020;21(7):935-946.
  7. AYVAKIT [prescribing information]. Cambridge, MA: Blueprint Medicines Corporation; May 2023.

INDICATION

AYVAKIT® (avapritinib) is indicated for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations.

INDICATION & IMPORTANT SAFETY INFORMATION

IMPORTANT SAFETY INFORMATION

Intracranial Hemorrhage — Serious intracranial hemorrhage (ICH) may occur with AYVAKIT treatment; fatal events occurred in <1% of patients. Overall, ICH (e.g., subdural hematoma, ICH, and cerebral hemorrhage) occurred in 2.9% of 749 patients who received AYVAKIT in clinical trials. In GIST patients, ICH occurred in 3 of 267 patients (1.1%) and two (0.7%) of the events were Grade ≥3 and resulted in discontinuation.

Monitor 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 thrombocytopenia. Symptoms of ICH may include headache, nausea, vomiting, vision changes, or altered mental status. Advise patients to seek immediate medical attention for signs or symptoms of ICH. Permanently discontinue AYVAKIT if ICH of any grade occurs.

Cognitive Effects — Cognitive adverse reactions can occur in patients receiving AYVAKIT and occurred in 33% of 995 patients overall in patients who received AYVAKIT in clinical trials including: 41% of 601 GIST patients (5% were Grade ≥3). Depending on the severity, withhold AYVAKIT and then resume at same dose or at a reduced dose upon improvement, or permanently discontinue.

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 of AYVAKIT. Advise women not to breastfeed during treatment with AYVAKIT and for 2 weeks following the final dose.

Adverse Reactions — The most common adverse reactions (≥20%) were edema, nausea, fatigue/asthenia, cognitive impairment, vomiting, decreased appetite, diarrhea, increased lacrimation, abdominal pain, constipation, rash, dizziness, and hair color changes.

Drug Interactions — Avoid coadministration of AYVAKIT with strong or moderate CYP3A inhibitors. If coadministration with a moderate CYP3A inhibitor cannot be avoided, reduce dose of AYVAKIT. Avoid coadministration of AYVAKIT with strong or moderate CYP3A inducers. If contraception requires estrogen, limit ethinyl estradiol to ≤20 mcg unless a higher dose is necessary.

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.

AYVAKIT is available in 100-mg, 200-mg and 300-mg tablets.

Please click here to see the full Prescribing Information for AYVAKIT.

References:

  1. Sun Y, Yue L, Xu P, Hu W. An overview of agents and treatments for PDGFRA-mutated gastrointestinal stromal tumors. Front Oncol. 2022;12:927587.
  2. Stamatakos M, Douzinas E, Stefanaki C, et al. Gastrointestinal stromal tumor. World J Surg Oncol. 2009;7:61.
  3. Akahoshi K, Oya M, Koga T, Shiratsuchi Y. Current clinical management of gastrointestinal stromal tumor. World J Gastroenterol. 2018;24(26):2806-2817.
  4. American Cancer Society. Gastrointestinal stromal tumor early detection, diagnosis, and staging. Accessed August 16, 2024. https://www.cancer.org/cancer/types/gastrointestinal-stromal-tumor/detection-diagnosis-staging.html
  5. Nishida T, Blay JY, Hirota S, Kitagawa Y, Kang YK. The standard diagnosis, treatment, and follow-up of gastrointestinal stromal tumors based on guidelines. Gastric Cancer. 2016;19(1):3-14.
  6. Heinrich MC, Jones RL, von Mehren M, et al. Avapritinib in advanced PDGFRA D842V-mutant gastrointestinal stromal tumour (NAVIGATOR): a multicentre, open-label, phase 1 trial. Lancet Oncol. 2020;21(7):935-946.
  7. AYVAKIT [prescribing information]. Cambridge, MA: Blueprint Medicines Corporation; May 2023.

The information on this website is intended solely for use by US payers, formulary committees, or other similar entities. These materials are not intended to be used by healthcare practitioners for the purpose of making individual patient prescribing or treatment decisions.

No, I am not a US formulary
decision-maker