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Voxelotor - Global Blood Therapeutics

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Drug Profile

Voxelotor - Global Blood Therapeutics

Alternative Names: GBT-440; GTx-011; Oxbryta

Latest Information Update: 06 Dec 2024

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At a glance

  • Originator Global Blood Therapeutics
  • Developer Global Blood Therapeutics; Pfizer
  • Class Antianaemics; Antifibrotics; Benzaldehydes; Ethers; Pyrazoles; Pyridines; Small molecules
  • Mechanism of Action Sickle haemoglobin inhibitors
  • Orphan Drug Status

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

    Yes - Sickle cell anaemia
  • New Molecular Entity Yes

Highest Development Phases

  • Registered Sickle cell anaemia
  • Discontinued Hypoxia; Idiopathic pulmonary fibrosis

Most Recent Events

  • 01 Nov 2024 Global Blood Therapeutics terminates an open-label extension HOPE trial in Sickle cell anaemia (In adolescents, In children) in Egypt, Lebanon, Nigeria, United Kingdom and USA (PO,Tablet,Powder) as the clinical data evaluated by Pfizer and shared with regulatory authorities indicates that the risk profile of voxelotor in people with SCD exceeds the benefits observed in previously generated global research and requires further assessment(NCT04188509)
  • 01 Oct 2024 Withdrawn for Sickle cell anaemia (In children, In the elderly, In adults, In adolescents) in United Arab Emirates (PO)
  • 30 Sep 2024 Withdrawn for Sickle cell anaemia (In adolescents, In adults) in United Kingdom (PO)

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