This site is intended for U.S. healthcare professionals.

Visit Pfizer Medical

Menu

Close

Sign InLog Out
ProductsOrderMaterialsCo-pay Cards & Patient Savings OffersRequest SamplesHospital ProductsVaccinesPatient AssistancePfizer Oncology TogetherPfizer RxPathwaysExplore ContentEventsMaterialsVideosContact

Menu

Close

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 adult and pediatric patients ages 12 years and older with sickle cell disease (SCD) in the Phase 3 HOPE trial1Oxbryta tablets were studied in a multicenter, randomized, double-blind, placebo-controlled (Phase 3) trial of 274 patients aged 12-64 years.

Patients were randomized to receive Oxbryta tablets 1,500 mg/day (n=90), Oxbryta tablets
900 mg/day (n=92), or placebo (n=92). Randomization was stratified by background hydroxyurea (HU) use, geographic region, and age.
Scroll left to view table
Primary Endpoint1 Select Secondary Endpoints1,2
  • Percentage of patients who had a hemoglobin (Hb) response (defined as an increase of >1 g/dL) from baseline to
    week 24
  • Change in Hb level from baseline at
    week 24
 
  • Change in laboratory markers associated with hemolysis (indirect bilirubin level and percentage of reticulocytes) from baseline at week 24
Study design detailsPatient Details

Select inclusion criteria1

  • Baseline Hb of 5.5-10.5 g/dL
  • 1-10 vaso-occlusive crisis (VOC) events within the last 12 months
  • Eligible patients on stable doses of HU for at least 90 days were allowed to continue HU therapy throughout the study

Select exclusion criteria1

  • Red blood cell transfusions within 60 days of enrollment
  • Erythropoietin use within 28 days of enrollment
  • Uncontrolled liver disease
  • Pregnant or lactating

Baseline demographics for patients in the Phase 3 HOPE trial1

Scroll left to view table
Characteristic Patients
Receiving background HU therapy 65%
Geographic region North America, Europe, other
Median age 24 years (12 to 64 years)
HbSS or HbSβ0 thalassemia genotype 90%
Pediatric patients 12 to <17 years 17%
Median baseline Hb 8.5 g/dL (5.9 to 10.8 g/dL)
1 VOC event in 12 months prior to enrollment 42%
2–10 VOC events in 12 months prior to enrollment 58%
HbSS = homozygous hemoglobin S; HbSβ0 = hemoglobin sickle beta zero.

Responders at 24 weeks

Hb Over 72 weeks (prespecified exploratory)

Oxbryta increased Hb levels in the Phase 3 HOPE trial1,2

51% of patients (from the intent-to-treat* population) receiving Oxbryta had a
>1 g/dL increase in Hb (compared to baseline) vs 7% receiving placebo (P<0.001).

Hb response rate at week 24 (intent-to-treat population; primary endpoint)1,*

All patients who were randomized into the study were included in the intent-to-treat population. Patients were analyzed based upon the treatment group to which they were assigned at randomization.3Results from a multicenter, randomized, double-blind, placebo-controlled parallel group study of 274 patients, aged 12-64 years, with SCD (a majority with HbSS or HbSβ0 thalassemia), conducted in 3 groups of study participants over a 24-week period.1,3

Hb responses in the per-protocol population1-3

Per-protocol analyses provide an estimate of efficacy but may not represent the circumstances seen in clinical practice, as these analyses are limited by reduced sample size and bias due to potential differential exclusion. Results are presented for descriptive purposes and offer supportive, but not conclusive, information.

Change in Hb for individual patients at week 24 (per-protocol population; secondary endpoint)1,†

59% of patients (from the per-protocol† population) receiving Oxbryta had a >1 g/dL increase in Hb (compared to baseline); 10% of patients receiving placebo2,3

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

  • Analyses were not prespecified or alpha protected (controlled for type 1 error); results are presented for descriptive purposes and offer supporting, but not conclusive, information
  • 42% (n=31/74) of patients receiving Oxbryta had a >1.5 g/dL increase in Hb (compared to baseline); 3% (n=2/76) of patients receiving placebo
  • 27% (n=20/74) of patients receiving Oxbryta had a >2.0 g/dL increase in Hb (compared to baseline); 1% (n=1/76) of patients receiving placebo

The per-protocol population included those randomized patients who completed 24 weeks of study drug and adhered to defined trial protocols (approximately 83% of all randomized patients).1,3Results from a multicenter, randomized, double-blind, placebo-controlled parallel group study of 274 patients, aged 12-64 years, with SCD (a majority with HbSS or HbSβ0 thalassemia), conducted in 3 groups of study participants over a 24-week period.1,3

Mean change in Hb from baseline through week 72 in the HOPE trial3,4

The results beyond week 24 are from a prespecified exploratory analysis and are not included in the Oxbryta USPI. Results are presented for descriptive purposes and offer supportive, but not conclusive, information.

The change from baseline to week 24 represents a secondary endpoint and the change from baseline to week 72 was a prespecified exploratory endpoint2,4

SELECTED SAFETY INFORMATION
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.

Hb response from baseline stratified by HU use3Results at 24 weeks and 72 weeks, stratified by baseline HU use

These results are from an exploratory analysis and are not included in the Oxbryta USPI. Subgroups were prespecified but not 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.

Adjusted mean change in Hb from baseline by background HU use

Indirect Bilirubin

Percent Reticulocytes

Oxbryta reduced hemolysis in the Phase 3 HOPE trial1,4

LS mean change from baseline in indirect bilirubin at week 24 (secondary endpoint)1,3

LS mean change from baseline in indirect bilirubin at week 723,4

The results at week 72 are from a prespecified exploratory analysis and are not included in the Oxbryta USPI. Results are presented for descriptive purposes and offer supportive, but not conclusive, information.

  • -23.9% for patients receiving Oxbryta (n=53)
  • 2.7% for patients receiving placebo (n=52)

Results from a multicenter, randomized, double-blind, placebo-controlled, parallel group study of 274 patients, aged 12-64, with SCD (a majority with HbSS or HbSß0 thalassemia), conducted in 3 groups of study participants over a 24-week period (pivotal analysis) and a 72-week period (prespecified end-of-study endpoint).1,2,4

LS = least squares.

Oxbryta reduced hemolysis in the Phase 3 HOPE trial1-4LS mean change from baseline in percent reticulocyte count at week 24 (secondary endpoint)1,3

LS mean change from baseline in percent reticulocyte count at week 723,4

The results at week 72 are from a prespecified exploratory analysis and are not included in the Oxbryta USPI. Results are presented for descriptive purposes and offer supportive, but not conclusive, information.

  • -7.6% for patients receiving Oxbryta (n=57)
  • 11.0% for patients receiving placebo (n=57)

Results from a multicenter, randomized, double-blind, placebo-controlled, parallel group study of 274 patients, aged 12-64, with SCD (a majority with HbSS or HbSβ0 thalassemia), conducted in 3 groups of study participants over a 24-week period (pivotal analysis) and a 72-week period (prespecified end-of-study endpoint).1,2,4

LS = least squares.

 Next: Clinical Data | Ages 4 to <12 Years Ages 4 to <12 years 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 EfficacySee Phase 3 HOPE trial results published in NEJM Learn more Loading 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

To report an adverse event, please call 1-800-438-1985

Pfizer for Professionals 1-800-505-4426

This site is intended only for U.S. healthcare professionals. The products discussed in this site may have different product labeling in different countries. The information provided is for educational purposes only.

© 2024 Pfizer Inc. All rights reserved.

PP-LTV-USA-2392
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).

You are now leaving PfizerPro You are now leaving a Pfizer operated website. Links to all outside sites are provided as a resource to our visitors. Pfizer accepts no responsibility for the content of sites that are not owned and operated by Pfizer. 
PP-MCL-USA-0367