Pharmacokinetics and pharmacodynamics of voxelotor (GBT440) in healthy adults and patients with sickle cell disease

A Hutchaleelaha, M Patel, C Washington… - British journal of …, 2019 - Wiley Online Library
Aims Voxelotor (previously GBT440) is a haemoglobin (Hb) modulator that increases Hb‐
oxygen affinity, thereby reducing Hb polymerization and sickling of red blood cells (RBCs) …

A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease

J Howard, CJ Hemmaway, P Telfer… - Blood, The Journal …, 2019 - ashpublications.org
New treatments directly targeting polymerization of sickle hemoglobin (HbS), the proximate
event in the pathophysiology of sickle cell disease (SCD), are needed to address the severe …

[HTML][HTML] A phase 3 randomized trial of voxelotor in sickle cell disease

E Vichinsky, CC Hoppe, KI Ataga… - … England Journal of …, 2019 - Mass Medical Soc
Abstract Background Deoxygenated sickle hemoglobin (HbS) polymerization drives the
pathophysiology of sickle cell disease. Therefore, direct inhibition of HbS polymerization has …

Voxelotor for the treatment of sickle cell disease

M Vissa, E Vichinsky - Expert Review of Hematology, 2021 - Taylor & Francis
Introduction: The clinical manifestations of sickle cell disease (SCD) result from an inherited
mutation in the beta-globin chain of hemoglobin (Hb) that causes Hb tetramers to polymerize …

Voxelotor: first approval

HA Blair - Drugs, 2020 - Springer
Voxelotor (Oxbryta™) is a haemoglobin S polymerization inhibitor that has been developed
for the treatment of sickle cell disease. In November 2019, voxelotor received its first global …

Voxelotor: a novel treatment for sickle cell disease

LB Herity, DMM Vaughan… - Annals of …, 2021 - journals.sagepub.com
Objective: To review the pharmacological characteristics, clinical evidence, and place in
therapy of voxelotor for the treatment of sickle cell disease (SCD). Data Sources: A …

Voxelotor in adolescents and adults with sickle cell disease (HOPE): long-term follow-up results of an international, randomised, double-blind, placebo-controlled …

J Howard, KI Ataga, RC Brown, M Achebe… - The Lancet …, 2021 - thelancet.com
Background For decades, patients with sickle cell disease have had only a limited number of
therapies available. In 2019, voxelotor (1500 mg), an oral once-daily sickle haemoglobin …

Voxelotor: alteration of sickle cell disease pathophysiology by a first-in-class polymerization inhibitor

AK Glaros, R Razvi, N Shah… - Therapeutic advances in …, 2021 - journals.sagepub.com
Sickle cell disease, despite its recognition as a severely debilitating genetic condition
affecting hundreds of thousands of neonates throughout the world each year, was not a …

Safety and efficacy of voxelotor in pediatric patients with sickle cell disease aged 4 to 11 years

JH Estepp, R Kalpatthi, G Woods… - Pediatric blood & …, 2022 - Wiley Online Library
Background Sickle cell disease (SCD) is a devastating, multisystemic disorder that affects
millions of people worldwide. The earliest clinical manifestations of SCD can affect infants as …

GBT 440 increases haemoglobin oxygen affinity, reduces sickling and prolongs RBC half‐life in a murine model of sickle cell disease

D Oksenberg, K Dufu, MP Patel… - British journal of …, 2016 - Wiley Online Library
A major driver of the pathophysiology of sickle cell disease (SCD) is polymerization of
deoxygenated haemoglobin S (HbS), which leads to sickling and destruction of red blood …