Voxelotor - Global Blood Therapeutics

Drug Profile

Voxelotor - Global Blood Therapeutics

Alternative Names: GBT-440; GTx-011

Latest Information Update: 11 Jul 2018

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At a glance

  • Originator Global Blood Therapeutics
  • Class Antianaemics; Small molecules
  • Mechanism of Action Sickle haemoglobin modulators
  • 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

  • Phase III Sickle cell anaemia
  • Phase I Liver disorders
  • Discontinued Hypoxia; Idiopathic pulmonary fibrosis

Most Recent Events

  • 27 Jun 2018 Efficacy and safety data from a phase III HOPE trial in Sickle cell disease released by Global Blood Therapeutics
  • 14 Jun 2018 Safety, pharmacokinetics and efficacy data from the phase IIa HOPE-KIDS 1 trial in Sickle cell anaemia presented at the 23rd European Haematology Association Congress (EHA-2018)
  • 06 Jun 2018 Global Blood Therapeutics initiates enrolment in a phase III trial for Sickle cell anaemia (In adolescents, In children, In adults) in USA (NCT03573882)
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