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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).
Contraindications
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.
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 (≥10%) in patients receiving 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 with sensitive CYP3A4 substrates with a narrow therapeutic index. If unavoidable, consider dose reduction of the 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 not to breastfeed while taking 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.
References: 1. Oxbryta Full Prescribing Information. South San Francisco, CA: Global Blood Therapeutics, Inc.; 12/2021. 2. 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.
References: 1. Oxbryta Full Prescribing Information. South San Francisco, CA: Global Blood Therapeutics, Inc.; 12/2021. 2. 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. 3. Kato GJ, Steinberg MH, Gladwin MT. lntravascular hemolysis and the pathophysiology of sickle cell disease. J Clin Invest. 2017;127(3):750-760. 4. Stuart MJ, Nagel RL. Sickle-cell disease. Lancet. 2004;364(9442):1343-1360. 5. Gordeuk VR, Castro OL, Machado RF. Pathophysiology and treatment of pulmonary hypertension in sickle cell disease. Blood. 2016;127(7):820-828. 6. Kapoor S, Little JA, Pecker LH. Advances in the treatment of sickle cell. Mayo Clin Proc. 2018;93(12):1810-1824. 7. Howard J. Hemmaway CJ, Telfer P, et al. A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease. Blood . 2019;133(17):1865-1875. 8. Data on file, Global Blood Therapeutics.
References: 1. Oxbryta Full Prescribing Information. South San Francisco, CA: Global Blood Therapeutics, Inc.; 12/2021. 2. 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. 3. Data on file, Global Blood Therapeutics. 4. Howard J, Ataga KJ, Brown RC, et al. Voxeletor 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)00059-4. 5. Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease (HOPE Kids). ClinicalTrials.gov identifier: NCT02850406. Updated May 13, 2021. Accessed August 16, 2021. https://clinicaltrials.gov/ct2/show/NCT02850406
References: 1. Oxbryta Full Prescribing Information. South San Francisco, CA: Global Blood Therapeutics, Inc.; 12/2021. 2. Data on file, Global Blood Therapeutics. 3. 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 Haemotol . 2021;8(5):e323-e333. doi:10.1016/S2352-3026(21)00059-4 4. Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease (HOPE Kids). ClinicalTrials.gov identifier: NCT02850406. Updated May 13, 2021. Accessed August 16, 2021. https://clinicaltrials.gov/ct2/show/NCT02850406
Reference: 1. Oxbryta Full Prescribing Information. South San Francisco, CA: Global Blood Therapeutics, Inc.; 12/2021.
Approved for patients as young as 4 years old1
