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SCD Root CauseAbout OxbrytaEfficacyEfficacyClinical Data | Ages ≥12 YearsClinical Data | Ages 4 to <12 YearsPatient ProfilesSafetyDosingAccess & SupportAccess & SupportAccess & SupportSign up
IndicationFull Prescribing InformationPatient Prescribing Information andInstructions for UsePatient Site
Oxbryta® (voxelotor) directly inhibits HbS polymerization to improve anemia and reduce hemolysis1,2Ages ≥12 YearsAges 4 to <12 YearsOxbryta was studied in pediatric patients ages 4 to <12 years with sickle cell disease (SCD) in the Phase 2 HOPE‑KIDS 1 trial1,5Studied in pediatric patients ages 4 to <12 years with sickle cell disease (SCD) in the Phase 2 HOPE-KIDS 1 trial1,5

Oxbryta was studied in an open-label, multicenter, Phase 2 study of 45 patients.

Patients aged 4 to <12 years received Oxbryta (voxelotor) tablets for oral suspension based on body weight at baseline.

  • Doses of 600 mg, 900 mg, or 1,500 mg once daily were administered to patients weighing 10 kg to <20 kg, 20 kg to <40 kg, or ≥40 kg, respectively
  • 35 patients received Oxbryta for 24 weeks
  • 26 patients received Oxbryta for 48 weeks
Efficacy Outcome1,5
  • Percentage of patients who achieved a response, defined as a Hb increase of >1 g/dL from baseline to week 24
Study design details

Patient Details

Select inclusion criteria1

  • Baseline Hb of ≤10.5 g/dL
  • Eligible patients on stable doses of hydroxyurea ≥90 days were allowed to continue hydroxyurea therapy throughout the study

Select exclusion criteria1

  • Patients who had a vaso-occlusive crisis event within 14 days prior to enrollment
  • Patients who received red blood cell transfusions within 30 days of enrollment
  • Patients with renal insufficiency or uncontrolled liver disease

Select baseline demographics1

  • Median age was 8 years (range: 4-15 years)*
    • 45 (80%) of patients were 4 to <12 years of age*
  • 80% received background hydroxyurea therapy*
  • Patients had a mean baseline of 8.6 g/dL (range: 6.1-10.5 g/dL)
  • All patients had HbSS or HbSβ0 thalassemia genotype (100%)
HOPE-KIDS 1 also included 11 patients aged 12 to <17 years who received Oxbryta 1,500 mg tablets once daily.1,5 Oxbryta significantly increased Hb levels in the HOPE-KIDS 1 trial1,3,5

36% of patients (from the intent-to-treat* population) receiving Oxbryta had a Hb increase of >1 g/dL from baseline to week 24 (95% CI: 21.6%, 49.5%)1,5

47% of patients (from the per-protocol† population) receiving Oxbryta had a Hb increase of >1 g/dL from baseline to week 24 (95% CI: 29.8%, 64.9%)3,5

Results from the per-protocol population are not included in the Oxbryta USPI. The per-protocol analysis was not prespecified or alpha protected (controlled for type 1 error), and hierarchical testing was not conducted. Results are presented for descriptive purposes and offer supportive, but not conclusive, information.

 Change in Hb for individual patients 4 to <12 at week 24 (per-protocol population)5

Post hoc analyses at week 24 in the per protocol population3,†

  • 35% (n=12/34) of patients receiving Oxbryta had a >1.5 g/dL increase in Hb from baseline3
  • 21% (n=7/34) of patients receiving Oxbryta had a >2.0 g/dL increase in Hb from baseline3
Results from an open-label, multicenter, Phase 2 study of 45 patients with SCD. Patients aged 4 to <12 years received Oxbryta tablets for oral suspension based on body weight at baseline. Doses of 600 mg, 900 mg, or 1,500 mg once daily were administered to patients weighing 10 kg to <20 kg, 20 kg to <40 kg, or ≥40 kg, respectively. All data presented in this section from the per-protocol analysis unless otherwise indicated.1,3Intent-to-treat: Included all patients who enrolled and received at least 1 dose of study drug regardless of adherence to defined trial protocols.3Per-protocol: Included patients who completed 24 weeks of study drug and had a Hb measurement available at week 24.3Next: Patient Profiles Patient profiles LoadingReferences:Oxbryta Full Prescribing Information. South San Francisco, CA: Global Blood Therapeutics, Inc.; 08/2023.Vichinsky E, Hoppe CC, Ataga Kl, et al. A phase 3 randomized trial of voxelotor in sickle cell disease. N Engl J Med. 2019;381(6):509-519. doi:10.1056/NEJMoa1903212Data on file. Pfizer Inc.Howard J, Ataga KI, Brown RC, et al. Voxelotor in adolescents and adults with sickle cell disease (HOPE): long-term follow-up results of an international, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Haematol. 2021;8(5):e323-e333. doi:10.1016/S2352-3026(21)00059-4Study to evaluate the effect of GBT440 in pediatrics with sickle cell disease (HOPE Kids). ClinicalTrials.gov identifier: NCT02850406. Updated January 19, 2022. Accessed February 24, 2022. https://clinicaltrials.gov/ct2/show/NCT02850406More on Efficacy Discover which of your SCD patients might be appropriate for Oxbryta Learn more Loading 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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