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Avapritinib - Blueprint Medicines

Drug Profile

Avapritinib - Blueprint Medicines

Alternative Names: AYVAKIT; AYVAKYT; BLU-285; CS 3007

Latest Information Update: 28 Feb 2024

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At a glance

  • Originator Blueprint Medicines
  • Developer Blueprint Medicines; CStone Pharmaceuticals; Neopharm Ltd
  • Class 2 ring heterocyclic compounds; Amines; Antineoplastics; Pyrazoles; Pyrimidines; Pyrroles; Skin disorder therapies; Small molecules; Triazines
  • Mechanism of Action Platelet derived growth factor alpha receptor antagonists; Proto oncogene protein c-kit inhibitors
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Systemic mastocytosis; Gastrointestinal stromal tumours
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Gastrointestinal stromal tumours; Systemic mastocytosis
  • Phase II Haematological malignancies
  • Phase I/II Solid tumours

Most Recent Events

  • 26 Feb 2024 Updated efficacy and adverse events data from the phase II PIONEER trial in Systemic Mastocytosis presented at the The American Academy of Allergy, Asthma & Immunology annual meeting (AAAAI annual meeting-2024)
  • 08 Jan 2024 Blueprint Medicines announces intention to launch avapritinib for systemic mastocytosis in Europe in 2024
  • 31 Dec 2023 Blueprint Medicines has patent pending for avapritinib worldwide
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