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: 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 …

[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 …

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 …

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 …

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 …

Real-world effectiveness of voxelotor for treating sickle cell disease in the US: a large claims data analysis

N Shah, T Lipato, O Alvarez, T Delea… - Expert review of …, 2022 - Taylor & Francis
Background Sickle cell disease (SCD) is a genetic disease that impacts patients' quality of
life, healthcare costs, and life expectancy. Elevated sickle hemoglobin (HbS), which readily …

[HTML][HTML] Efficacy and safety of recently approved drugs for sickle cell disease: a review of clinical trials

MA Ali, A Ahmad, H Chaudry, W Aiman, S Aamir… - Experimental …, 2020 - Elsevier
Highlights•VOC episodes and low hemoglobin levels are major complications of SCD.•L-
Glutamine decreases the episodes of VOC.•Voxelotor increases hemoglobin levels and …

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) …

Real‐life experience with hydroxyurea in patients with sickle cell disease: results from the prospective ESCORT‐HU cohort study

M de Montalembert, E Voskaridou… - American journal of …, 2021 - Wiley Online Library
Several controlled studies have evidenced good efficacy and short‐term and mid‐term
safety profiles for hydroxyurea (HU), which has become the cornerstone for prevention of …