A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease
Jun 13, 2019 · Voxelotor is an HbS polymerization inhibitor. In a multicenter, phase 3, double-blind, randomized, placebo-controlled trial, we compared the efficacy and safety of two dose levels of...
Practical Guidance for the Use of Voxelotor in the Management of Sickle …
Expected clinical efficacy is a key consideration in prescribing voxelotor for SCD. Clinical trials demonstrated statistically significant benefits related to hemoglobin levels and markers of hemolysis, while impact on clinical outcomes varied.
Efficacy and Safety of Voxelotor in Sickle Cell Disease: A …
Nov 5, 2020 · Voxelotor has an acceptable safety profile in sickle cell disease patients of 12 years or older. Voxelotor has shown a dose-dependent improvement in hemoglobin levels and markers of hemolysis, which is associated with a reduction in end-organ damage.
Voxelotor: A Hemoglobin S Polymerization Inhibitor for the …
Nov 8, 2020 · Voxelotor may inhibit red blood cell sickling, improve red blood cell deformability, and reduce whole blood viscosity, which contributes to anemia and hemolysis (Global Blood Therapeutics, 2019).
FDA approves novel treatment to target abnormality in sickle cell disease
Today, the U.S. Food and Drug Administration granted accelerated approval to Oxbryta (voxelotor) for the treatment of sickle cell disease (SCD) in adults and pediatric patients 12 years of age...
Voxelotor: alteration of sickle cell disease pathophysiology by …
This review aims to summarize the targeted pathobiology of sickle cell disease, the mechanism of action of voxelotor, and the safety and efficacy data from preclinical to late clinical stage investigations of this long-awaited medication, in the hopes of better informing the decision-making process behind prescribing or not prescribing it for ...
Voxelotor in adolescents and adults with sickle cell disease …
Apr 7, 2021 · Voxelotor is a HbS polymerisation inhibitor that has been approved by the US Food and Drug Administration for the treatment of sickle cell disease in adult and adolescent patients (aged 12 years and older) at a dose of 1500 mg orally once daily.
Oxbryta (voxelotor) - Sickle Cell Disease News
May 22, 2024 · What is Oxbryta for sickle cell disease? Oxbryta (voxelotor) is an oral therapy conditionally approved in the U.S. for children and adults with sickle cell disease (SCD), 4 and older. It is used to prevent red blood cell sickling and destruction, and to reduce blood viscosity caused by the production of a faulty version of hemoglobin — the ...
Patient perception of voxelotor treatment benefit in sickle cell disease
Voxelotor is a novel sickle hemoglobin (HbS) polymerization inhibitor that has been shown to improve key measures of red blood cell (RBC) heath in patients with SCD. 8–10 In a phase III clinical trial, patients receiving voxelotor therapy showed rapid and durable improvements in hemoglobin (Hb) concentrations and markers of hemolysis. 11 12 ...
FDA approves drug to treat sickle cell disease in pediatric patients
Dec 17, 2021 · FDA has granted accelerated approval for Oxbryta (voxelotor) tablets to treat sickle cell disease in pediatric patients aged four up to 11 years.