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SCD Root CauseAbout OxbrytaEfficacyEfficacyClinical Data | Ages ≥12 YearsClinical Data | Ages 4 to <12 YearsPatient ProfilesSafetyDosingAccess & SupportAccess & SupportAccess & SupportSign up
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Hemoglobin S (HbS) polymerization is the root cause of red blood cell (RBC) sickling in sickle cell disease (SCD)1-5Hemoglobin S (HbS) polymerization is the root cause of red blood cell (RBC) sickling in sickle cell disease (SCD)1-5
  • HbS molecules have a lower affinity for oxygen
  • When HbS releases oxygen, it can polymerize into long, rigid rods
  • These long, rigid rods deform the RBCs into sickled RBCs which, in turn, may contribute to other complications of SCD
HbS polymerization leads to RBC sickling and may also contribute to other SCD complications, including organ damage due to1,5-7:HbS polymerization leads to RBC sickling and may also contribute to other SCD complications, including organ damage due to1,5-7:

While HbS polymerization in RBCs may occur in patients with SCD, the extent to which it contributes to the development of SCD complications, including organ damage, is unknown. Other genetic, environmental, and/or psychosocial factors may also affect the development of SCD complications in patients with SCD.8-10

Every patient with SCD experiences pathophysiology, such as anemia and hemolysis11Every patient with SCD experiences pathophysiology, such as anemia and hemolysis11

Anemia, hemolysis, vaso-occlusion, endothelial dysfunction, and inflammation are pathological hallmarks of SCD5,11

 Next: How Oxbryta works

Oxbryta is designed to specifically address the root cause of RBC sickling in SCD34

 How Oxbryta works LoadingReferences:Kato GJ, Steinberg MH, Gladwin MT. Intravascular hemolysis and the pathophysiology of sickle cell disease. J Clin Invest. 2017;127(3):750-760. doi:10.1172/JCI89741Stuart MJ, Nagel RL. Sickle-cell disease. Lancet. 2004;364(9442):1343-1360. doi:10.1016/S0140-6736(04)17192-4Gordeuk VR, Castro OL, Machado RF. Pathophysiology and treatment of pulmonary hypertension in sickle cell disease. Blood. 2016;127(7):820-828. doi:10.1182/blood-2015-08-618561Kapoor S, Little JA, Pecker LH. Advances in the treatment of sickle cell disease. Mayo Clin Proc. 2018;93(12):1810-1824. doi:10.1016/j.mayocp.2018.08.001Kato GJ, Piel FB, Reid CD, et al. Sickle cell disease. Nat Rev Dis Primers. 2018;4:18010. doi:10.1038/nrdp.2018.10Rees DC, Williams TN, Gladwin MT. Sickle-cell disease. Lancet. 2010;376(9757):2018-2031. doi:10.1016/S0140-6736(10)61029-XCaboot JB, Allen JL. Hypoxemia in sickle cell disease: significance and management. Paediatr Respir Rev. 2014;15(1):17-23. doi:10.1016/j.prrv.2013.12.004Sundd P, Gladwin MT, Novelli EM. Pathophysiology of sickle cell disease. Annu Rev Pathol. 2019;14:263-292. doi:10.1146/annurev-pathmechdis-012418-012838Tewari S, Brousse V, Piel FB, Menzel S, Rees DC. Environmental determinants of severity in sickle cell disease. Haematologica. 2015;100(9):1108-1116. doi:10.3324/haematol.2014.120030Vichinsky E. Chronic organ failure in adult sickle cell disease. Hematology Am Soc Hematol Educ Program. 2017;2017(1):435-439. doi:10.1182/asheducation-2017.1.435Nader E, Romana M, Connes P. The red blood cell—inflammation vicious circle in sickle cell disease. Front Immunol. 2020;11:454. doi:10.3389/fimmu.2020.00454Saraf S, Farooqui M, Infusino G, et al. Standard clinical practice underestimates the role and significance of erythropoietin deficiency in sickle cell disease. Br J Haematol. 2011;153(3):386-392. doi:10.1111/j.1365-2141.2010.08479.xAtaga KI, Gordeuk VR, Agodoa I, Colby JA, Gittings K, Allen IE. Low hemoglobin increases risk for cerebrovascular disease, kidney disease, pulmonary vasculopathy, and mortality in sickle cell disease: a systematic literature review and meta-analysis. PLoS One. 2020;15(4):e0229959. doi:10.1371/journal.pone.0229959Elmariah H, Garrett ME, De Castro LM, et al. Factors associated with survival in a contemporary adult sickle cell disease cohort. Am J Hematol. 2014;89(5):530-535. doi:10.1002/ajh.23683Chaturvedi S, Ghafuri DL, Jordan N, Kassim A, Rodeghier M, DeBaun MR. Clustering of end-organ disease and earlier mortality in adults with sickle cell disease: a retrospective-prospective cohort study. Am J Hematol. 2018;93(9):1153-1160. doi:10.1002/ajh.25202Damy T, Bodez D, Habibi A, et al. Haematological determinants of cardiac involvement in adults with sickle cell disease. Eur Heart J. 2016;37(14):1158-1167. doi:10.1093/eurheartj/ehv555Galadanci NA, Johnson W, Carson A, Hellemann G, Howard V, Kanter J. Factors associated with left ventricular hypertrophy in children with sickle cell disease: results from the DISPLACE study. Haematologica. 2022;107(10):2466-2473. doi:10.3324/haematol.2021.280480Ershler WB, De Castro LM, Pakbaz Z, et al. Hemoglobin and end-organ damage in individuals with sickle cell disease. Curr Ther Res Clin Exp. 2023;98:100696. doi:10.1016/j.curtheres.2023.100696Lebensburger J, Johnson SM, Askenazi DJ, Rozario NL, Howard TH, Hilliard LM. Protective role of hemoglobin and fetal hemoglobin in early kidney disease for children with sickle cell anemia. Am J Hematol. 2011;86(5):430-432. doi:10.1002/ajh.21994Nebor D, Broquere C, Brudey K, et al. Alpha-thalassemia is associated with a decreased occurrence and a delayed age-at-onset of albuminuria in sickle cell anemia patients. Blood Cells Mol Dis. 2010;45(2):154-158. doi:10.1016/j.bcmd.2010.06.003Ford AL, Ragan DK, Fellah S, et al. Silent infarcts in sickle cell disease occur in the border zone region and are associated with low cerebral blood flow. Blood. 2018;132(16):1714-1723. doi:10.1182/blood-2018-04-841247Salama K, Rady R, Hashem RH, El-Ghamrawy M. Transcranial Doppler velocities among sickle cell disease patients in steady state. Hemoglobin. 2020;44(6):418-422. doi:10.1080/03630269.2020.1843483Meier ER, Wright EC, Miller JL. Reticulocytosis and anemia are associated with an increased risk of death and stroke in the newborn cohort of the Cooperative Study of Sickle Cell Disease. Am J Hematol. 2014;89(9):904-906. doi:10.1002/ajh.23777Niss O, Quinn CT, Lane A, et al. Cardiomyopathy with restrictive physiology in sickle cell disease. JACC Cardiovasc Imaging. 2016;9(3):243-252. doi:10.1016/j.jcmg.2015.05.013Gladwin MT, Sachdev V, Jison ML, et al. Pulmonary hypertension as a risk factor for death in patients with sickle cell disease. N Engl J Med. 2004;350(9):886-895. doi:10.1056/NEJMoa035477Nouraie M, Lee JS, Zhang Y, et al. The relationship between the severity of hemolysis, clinical manifestations and risk of death in 415 patients with sickle cell anemia in the US and Europe. Haematologica. 2013;98(3):464-472. doi:10.3324/haematol.2012.068965Nouraie M, Darbari DS, Rana S, et al. Tricuspid regurgitation velocity and other biomarkers of mortality in children, adolescents and young adults with sickle cell disease in the United States: the PUSH study. Am J Hematol. 2020;95(7):766-774. doi:10.1002/ajh.25799Rosenberg JB, Hutcheson KA. Pediatric sickle cell retinopathy: correlation with clinical factors. J AAPOS. 2011;15(1):49-53. doi:10.1016/j.jaapos.2010.11.014Mesleh Shayeb A, Smeltzer MP, Kaste SC, Brown A, Estepp JH, Nottage KA. Vaso-occlusive crisis as a predictor of symptomatic avascular necrosis in children with sickle cell disease. Pediatr Blood Cancer. 2018;65(12):e27435. doi:10.1002/pbc.27435Cavalcante JEA, Machado RPG, Laurentino MR, et al. Clinical events and their relation to the tumor necrosis factor-alpha and interleukin-10 genotypes in sickle-cell-anemia patients. Hematol Oncol Stem Cell Ther. 2016;9(1):14-19. doi:10.1016/j.hemonc.2015.11.002Yousef AA, Shash HA, Almajid AN, et al. Predictors of recurrent acute chest syndrome in pediatric sickle cell disease: a retrospective case-control study. Children (Basel). 2022;9(6):894. doi:10.3390/children9060894Nawaiseh M, Roto A, Nawaiseh Y, et al. Risk factors associated with sickle cell retinopathy: findings from the Cooperative Study of Sickle Cell Disease. Int J Retina Vitreous. 2022;8(1):68. doi:10.1186/s40942-022-00419-8Zhang D, Xu C, Manwani D, Frenette PS. Neutrophils, platelets, and inflammatory pathways at the nexus of sickle cell disease pathophysiology. Blood. 2016;127(7):801-809. doi:10.1182/blood-2015-09-618538Oxbryta Full Prescribing Information. South San Francisco, CA: Global Blood Therapeutics, Inc.; 08/2023. Get the latest information about Oxbryta delivered to your inbox Sign up Loading

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Indications and UsageOxbryta is indicated for the treatment of sickle cell disease (SCD) in adults and pediatric patients 4 years of age and older.

This indication is approved under accelerated approval based on increase in hemoglobin (Hb). Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
Important Safety InformationCONTRAINDICATIONS
Oxbryta is contraindicated in patients with a history of serious drug hypersensitivity reaction to voxelotor or excipients. Clinical manifestations may include generalized rash, urticaria, mild shortness of breath, mild facial swelling, and eosinophilia.

WARNINGS AND PRECAUTIONS
Hypersensitivity Reactions
Serious hypersensitivity reactions after administration of Oxbryta have occurred in <1% of patients treated. Clinical manifestations may include generalized rash, urticaria, mild shortness of breath, mild facial swelling, and eosinophilia.

Drug reaction with eosinophilia and systemic symptoms (DRESS) has been reported in postmarketing experience with Oxbryta. Patients who develop a combination of skin rash, fever, peripheral eosinophilia, and internal systemic organ involvement (e.g., hepatic, renal, pulmonary) while receiving Oxbryta should undergo medical evaluation.

Advise patients of the signs and symptoms of severe hypersensitivity reactions, including DRESS. If hypersensitivity reactions occur, discontinue Oxbryta and administer appropriate medical therapy. Do not reinitiate Oxbryta in patients who experience these symptoms with previous use.

Laboratory Test Interference
Oxbryta administration may interfere with measurement of Hb subtypes (HbA, HbS, and HbF) by high-performance liquid chromatography (HPLC). If precise quantitation of Hb species is required, chromatography should be performed when the patient has not received Oxbryta therapy in the immediately preceding 10 days.

ADVERSE REACTIONS
Clinical Trials Experience
Adults and Pediatric Patients 12 Years of Age and Older
Serious adverse reactions occurred in 3% (3/88) of patients receiving Oxbryta 1,500 mg, which included headache, drug hypersensitivity, and pulmonary embolism occurring in 1 patient each. Permanent discontinuation due to an adverse reaction (Grades 1-4) occurred in 5% (4/88) of patients who received Oxbryta 1,500 mg.

The most common adverse reactions occurring in ≥10% of patients treated with Oxbryta 1,500 mg with a difference of >3% compared to placebo: Headache (32% vs. 25%), Diarrhea (23% vs. 11%), Abdominal Pain (23% vs. 16%), Nausea (19% vs. 10%), Rash (15% vs. 11%), and Pyrexia (15% vs. 8%).

Pediatric Patients 4 to <12 Years
The safety of Oxbryta in pediatric patients 4 to <12 years with SCD was evaluated in an open-label, Phase 2 study. In this study, 45 patients 4 to <12 years of age received doses of Oxbryta tablets for oral suspension based on weight at baseline. Thirty-five patients received Oxbryta for 24 weeks and 26 patients for 48 weeks. The most common adverse reactions (>10%) reported in pediatric patients 4 to <12 years were pyrexia (36%), vomiting (33%), rash (20%), abdominal pain (18%), diarrhea (18%), and headache (18%).

The overall safety profile of Oxbryta in pediatric patients 4 to <12 years was similar to that seen in adults and pediatric patients 12 years and older.

DRUG INTERACTIONS
Strong or Moderate CYP3A4 Inducers
Coadministration of strong or moderate CYP3A4 inducers may decrease voxelotor plasma and whole blood concentrations and may lead to reduced efficacy. Avoid coadministration of Oxbryta with strong or moderate CYP3A4 inducers. Increase the Oxbryta dosage when coadministration with a strong or moderate CYP3A4 inducer is unavoidable.

Sensitive CYP3A4 Substrates
Voxelotor increased the systemic exposure of midazolam (a sensitive CYP3A4 substrate). Avoid coadministration of Oxbryta with sensitive CYP3A4 substrates with a narrow therapeutic index. If concomitant use is unavoidable, consider dose reduction of the sensitive CYP3A4 substrate(s).

USE IN SPECIFIC POPULATIONS
Lactation
Because of the potential for serious adverse reactions in the breastfed child, including changes in the hematopoietic system, advise patients that breastfeeding is not recommended during treatment with Oxbryta, and for at least 2 weeks after the last dose.

Recommended Dosage for Hepatic Impairment
Severe hepatic impairment increases voxelotor exposures. For severe hepatic impairment (Child Pugh C) reduce dose to 1,000 mg orally once daily for adults and pediatric patients ≥12 years. Dose reduction for pediatric patients 4 to <12 years is dependent on body weight (please refer to Table 2 in the Full Prescribing Information).

Please see Full Prescribing Information for more information about Oxbryta.Indications and Usage

Oxbryta is indicated for the treatment of sickle cell disease (SCD) in adults and pediatric patients 4 years of age and older.

This indication is approved under accelerated approval based on increase in hemoglobin (Hb). Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).

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