CDEK
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avapritinib

Trade name: Ayvakit

Small moleculeapprovedOrphan Drug FDAFast Track FDABreakthrough Therapy FDA

Approved

Jan 9, 2020

Blu-285 is a potent and selective inhibitor for hematologic malignancies with KIT Exon 17 Mutations. BLU-285 has demonstrated biochemical in vitro activity on the KIT exon-17 mutant enzyme, KIT D816V. Cellular activity of BLU-285 on KIT D816 mutants was measured by autophosphorylation in the human mast cell leukemia cell line HMC1.2, and the P815 mouse mastocytoma cell line with IC50= 4 and 22 nM, respectively. In vivo BLU-285 was well tolerated and has demonstrated dose-dependent antitumor efficacy. Complete tumor growth inhibition and ≥ 75% KIT kinase inhibition was observed with 10 mg/kg once daily, oral dosing of BLU-285 in the aggressive KIT exon 17 mutant driven P815 mastocytoma model grown as a solid tumor allograft as well as in a disseminated model of the disease. BLU-285 was also well tolerated in this in vivo model and had no adverse effects on body weight at either dose. — NCATS

Clinical trial activity

21 trials · 10 clinical orgs · 1 marketing orgs

Phase 1
7
Phase 2
12
Phase 3
1
Phase 4
2

Earliest trial started Aug 1, 2015 (NCT02508532)

Timeline

2010s

  1. Jan 1, 2015

    Blueprint Medicines Corporation — Earliest Phase 1 Sponsor(trial)

  2. Jan 6, 2016

    Orphan Drug Designation

  3. Jan 1, 2018

    Blueprint Medicines Corporation — Earliest Phase 2 Sponsor(trial)

  4. Jan 1, 2018

    Blueprint Medicines Corporation — Earliest Phase 3 Sponsor(trial)

2020s

  1. Jan 9, 2020

    Blueprint Medicines Corporation — Earliest FDA Approval

  2. Jan 9, 2020

    Blueprint Medicines Corporation — Marketing Organization

  3. Jan 9, 2020

    Blueprint Medicines Corporation — NDA Secondary Org

  4. Jan 9, 2020

    Blueprint Medicines Corporation — NDA Organization

Indications

Approved for

Studied for

Mechanism of action

Approval history

  • approvedPriority reviewFast trackBreakthroughJan 9, 2020

Chemistry & pharmacology

Loading structure…

SMILES

CN1C=C(C=N1)C2=CN3N=CN=C(N4CCN(CC4)C5=NC=C(C=N5)[C@@](C)(N)C6=CC=C(F)C=C6)C3=C2
Mol. weight
498.558 g/mol
Lipinski Ro5
Pass
Rule of 3
No
Chirality
Single Stereoisomer
Inorganic
No
Polymer
No
Delivery
Oral
Availability
Prescription Only

Oral

Yes

Parenteral

No

Topical

No

Sources

Also known as

  • blu-285
  • blu-285

Clinical trials

NCT IDPhaseStart dateSponsor(s)
NCT07028073Phase 1/Phase 2 (Phase 2)May 15, 2025Soochow University
NCT06765915Phase 2Feb 1, 2025Jiao Tong University
NCT06783790Phase 2Jan 20, 2025China Academy of Chinese Medical Sciences
NCT06748001Phase 4Nov 28, 2024Blueprint Medicines Corporation
NCT07131059N/AMay 11, 2024China Academy of Chinese Medical Sciences, People's Liberation Army of China
NCT06327685Phase 1Mar 13, 2024Blueprint Medicines Corporation, University of South Florida
NCT06316960Phase 2Mar 1, 2024Soochow University
NCT06221683Phase 2Jan 1, 2024Soochow University
NCT05821738Phase 2Jun 1, 2022Soochow University
NCT04773782Phase 1/Phase 2 (Phase 2)Nov 30, 2021Blueprint Medicines Corporation
NCT04908176Phase 1Jul 31, 2021Blueprint Medicines Corporation
NCT04825574Phase 4May 21, 2021Blueprint Medicines Corporation
NCT04771520Phase 2Jan 20, 2021National Cancer Institute (NCI), University of Texas at Houston
NCT04254939Phase 1/Phase 2 (Phase 2)Aug 19, 2019Blueprint Medicines Corporation, Chinese Academy of Medical Sciences, CStone Pharmaceuticals
NCT03731260Phase 2Apr 16, 2019Blueprint Medicines Corporation
NCT03580655Phase 2Sep 30, 2018Blueprint Medicines Corporation
NCT03465722Phase 3Mar 26, 2018Blueprint Medicines Corporation
NCT02561988Phase 1Dec 1, 2015Blueprint Medicines Corporation
NCT02508532Phase 1Aug 1, 2015Blueprint Medicines Corporation
NCT03862885N/A—Blueprint Medicines Corporation
Showing 20 of 21 trials
Page 1 / 2

Organizations

Research & Development (10)

OrganizationOrg typeTrialsAs lead sponsorPhasesEarliest year
Blueprint Medicines CorporationFor profit131152015
Soochow UniversityAcademic/Hospital4412022
China Academy of Chinese Medical SciencesAcademic/Hospital2222024
Chinese Academy of Medical SciencesAcademic/Hospital1012019
CStone PharmaceuticalsFor profit1112019
Jiao Tong UniversityAcademic/Hospital1112025
National Cancer Institute (NCI)Government1012021
People's Liberation Army of ChinaGovernment1012024
University of South FloridaAcademic/Hospital1112024
University of Texas at HoustonAcademic/Hospital1112021
10 organizations
Page 1 / 1

Marketing (3)

OrganizationOrg typeRelationshipDate
Blueprint Medicines CorporationFor profitMKTGJan 9, 2020
Blueprint Medicines CorporationFor profitNDA2Jan 9, 2020
Blueprint Medicines CorporationFor profitNDAJan 9, 2020