Drugs / Avapritinib
last change May 2026 re-read 2 minutes ago

Avapritinib

also known as BLU-285 · 70C366 · Ayvakit · Ayvakyt · C-366 · X-720776

Small molecule targets KIT, PDGFRA via inhibition

Developed for
gastrointestinal stromal tumor · acute myeloid leukemia · acute myeloid leukemia, KIT gene mutation · systemic mastocytosis with an associated clonal hematologic non-mast cell lineage disease · aggressive systemic mastocytosis · cancer · central nervous system cancer · childhood acute myeloid leukemia
+19 more · core binding factor acute myeloid leukemia · mast cell leukemia · refractory malignant neoplasm · acute myeloid leukemia with t(8;21)(q22;q22) translocation · acute myeloid leukemia, t(16;16)(p13.1;q22) · central nervous system neoplasm · childhood malignant neoplasm · colorectal carcinoma · female breast carcinoma · gastroesophageal junction adenocarcinoma · high grade malignant neoplasm · indolent systemic mastocytosis · lung carcinoma · metastatic malignant neoplasm · metastatic melanoma · myeloproliferative neoplasm · sarcoma · soft tissue sarcoma · systemic mastocytosis
Investigated by
Blueprint Medicines Corporation · Children's Hospital of Soochow University · Institute of Hematology & Blood Diseases Hospital, China · The First Affiliated Hospital of Soochow University · CStone Pharmaceuticals · Centre Leon Berard
+9 more · Fred Hutchinson Cancer Center · H. Lee Moffitt Cancer Center and Research Institute · M.D. Anderson Cancer Center · National Cancer Institute (NCI) · Northwell Health · Oregon Health and Science University · Presage Biosciences · Ruijin Hospital · University of Washington

Regulatory milestones approvals, filings & regulatory actions · 4 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Label expansions (2) US (FDA) AYVAKIT 2021-06-16 – 2023-05-22 fda.gov
Approved · conditional EU (EMA) Ayvakyt 2020-09-24 europa.eu
Approved US (FDA) AYVAKIT 2020-01-09 fda.gov

Trials 22 · a red edge is where a trial was stopped

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 15 trials
Phase 1 NCT06327685 Mar 2024 → Mar 2027 expected systemic mastocytosis with an associated clonal hematologic non-mast cell lineage disease H. Lee Moffitt Cancer Center and Research Institute Recruiting No outcome recorded
Phase 1 NCT04908176 Aug 2022 → Mar 2024 central nervous system cancer, gastrointestinal stromal tumor Blueprint Medicines Corporation Completed No outcome recorded
Phase 1 NCT03056599 Dec 2016 → Jul 2021 soft tissue sarcoma Presage Biosciences Completed No outcome recorded
Phase 1 NCT02561988 Mar 2016 → Oct 2022 aggressive systemic mastocytosis, mast cell leukemia, myeloproliferative neoplasm, systemic mastocytosis with an associated clonal hematologic non-mast cell lineage disease Blueprint Medicines Corporation Completed No outcome recorded
Phase 1 NCT02508532 Aug 2015 → Mar 2020 gastrointestinal stromal tumor, refractory malignant neoplasm Blueprint Medicines Corporation Completed No outcome recorded
Phase 1/23 trials
Phase 1/2 NCT07028073 May 2025 → May 2027 expected acute myeloid leukemia with t(8;21)(q22;q22) translocation, acute myeloid leukemia, KIT gene mutation, acute myeloid leukemia, t(16;16)(p13.1;q22) The First Affiliated Hospital of Soochow University Recruiting No outcome recorded
Phase 1/2 NCT04773782 Feb 2022 → Nov 2025 central nervous system neoplasm, childhood malignant neoplasm, refractory malignant neoplasm Blueprint Medicines Corporation Completed No outcome recorded
Phase 1/2 NCT04254939 Aug 2019 → Apr 2020 gastrointestinal stromal tumor CStone Pharmaceuticals Completed No outcome recorded
Phase 29 trials
Phase 2 NCT06765915 Feb 2025 → Feb 2026 overdue acute myeloid leukemia Ruijin Hospital Not yet recruiting No outcome recorded
Phase 2 NCT06783790 Jan 2025 → Dec 2026 expected acute myeloid leukemia Institute of Hematology & Blood Diseases Hospital, China Recruiting No outcome recorded
Phase 2 NCT06316960 Mar 2024 → Mar 2026 overdue acute myeloid leukemia, KIT gene mutation, childhood acute myeloid leukemia, core binding factor acute myeloid leukemia Children's Hospital of Soochow University Recruiting No outcome recorded
Phase 2 NCT06221683 Jan 2024 → Dec 2028 expected childhood acute myeloid leukemia Children's Hospital of Soochow University Recruiting No outcome recorded
Phase 2 NCT05821738 Jun 2022 → Dec 2024 acute myeloid leukemia, KIT gene mutation, core binding factor acute myeloid leukemia The First Affiliated Hospital of Soochow University Unknown No outcome recorded
Phase 2 NCT04771520 Jan 2021 → Dec 2026 expected central nervous system cancer, colorectal carcinoma, female breast carcinoma, gastroesophageal junction adenocarcinoma +5 M.D. Anderson Cancer Center Recruiting No outcome recorded
Phase 2 NCT04116541 Jan 2020 → Oct 2026 expected cancer Centre Leon Berard Recruiting No outcome recorded
Phase 2 NCT03731260 Apr 2019 → Jun 2027 expected indolent systemic mastocytosis Blueprint Medicines Corporation Active not recruiting No outcome recorded
Phase 2 NCT03580655 Nov 2018 → Dec 2024 aggressive systemic mastocytosis, mast cell leukemia, systemic mastocytosis with an associated clonal hematologic non-mast cell lineage disease Blueprint Medicines Corporation Completed No outcome recorded
Phase 31 trial
Phase 3 NCT03465722 Mar 2018 → Mar 2020 gastrointestinal stromal tumor Blueprint Medicines Corporation Completed No outcome recorded
Phase 42 trials
Phase 4 NCT06748001 Nov 2024 → Dec 2027 expected systemic mastocytosis Blueprint Medicines Corporation Recruiting No outcome recorded
Phase 4 NCT04825574 May 2021 → Nov 2023 gastrointestinal stromal tumor Blueprint Medicines Corporation Completed No outcome recorded
Phase not applicable1 trial
NCT07131059 May 2024 → May 2026 overdue acute myeloid leukemia Institute of Hematology & Blood Diseases Hospital, China Recruiting No outcome recorded
Phase not stated1 trial
NCT04714086 cancer Blueprint Medicines Corporation No longer available No outcome recorded
Also used as a comparator or background therapy in 1 trial
PhaseRegistry idDatesIndicationSponsorStatusOutcome
NCT03862885 Background gastrointestinal stromal tumor Blueprint Medicines Corporation Approved for marketing No outcome recorded

Evidence & citations 12 cited values

Every value below carries the sentence it was read from. 26 sources stand behind the page.

FieldValueCited text
Known as Avapritinib Avapritinib is a type 1 kinase inhibitor designed to potently and selectively inhibit oncogenic KIT/PDGFRA mutants by targeting the kinase active conformation.” PMID 36477870 Feb 2023
22

“Drug: AvapritinibNCT04116541

NCT05821738

NCT02508532

NCT04771520

NCT02561988

NCT06327685

NCT03056599

NCT07131059

NCT03731260

NCT04908176

NCT07028073

NCT06316960

NCT03862885

NCT06748001

NCT04825574

NCT04773782

NCT06783790

NCT06221683

NCT06765915

NCT03465722

NCT04714086

NCT03580655

Known as 70C366 ChEMBL registry synonym — accepted as the source's own label CHEMBL4204794
Known as Ayvakit ChEMBL registry synonym — accepted as the source's own label CHEMBL4204794
Known as Ayvakyt ChEMBL registry synonym — accepted as the source's own label CHEMBL4204794
Known as BLU-285 “This phase 1 study ( NCT02561988 ) evaluated avapritinib (BLU-285), a selective KIT D816V inhibitor, in patients with AdvSM.” PMID 34873347 Dec 2021
1

“This record comprises the currently available Expanded Access Programs (EAP) for avapritinib (BLU-285):” NCT04714086

Known as C-366 ChEMBL registry synonym — accepted as the source's own label CHEMBL4204794
Known as X-720776 ChEMBL registry synonym — accepted as the source's own label CHEMBL4204794
Action Inhibit “Avapritinib selectively and potently inhibits KIT- and PDGFRA-mutant kinases.” PMID 34343033 Aug 2021
Modality Small molecule “a small molecular targeted agent (Avapritinib)” NCT07028073
Route Oral “administered orally (PO)” NCT02561988
Target KIT “Avapritinib selectively and potently inhibits KIT- and PDGFRA-mutant kinases.” PMID 34343033 Aug 2021
Target PDGFRA “Avapritinib selectively and potently inhibits KIT- and PDGFRA-mutant kinases.” PMID 34343033 Aug 2021