Substance / Medication

Voxelotor

Overview

Active Ingredient
voxelotor
RxNorm CUI
2265678

Indications

OXBRYTA is indicated for the treatment of sickle cell disease (SCD) in adults and pediatric patients 4 years of age and older. Clinical Studies (14) [see] 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).

Labeler: Global Blood Therapeutics, Inc, A subsidiary of Pfizer Inc.Updated: 2023-12-26T00:00:00.000ZFull label on DailyMed

Contraindications

When this intervention should not be used

Warnings and Precautions (5.1) Adverse Reactions (6.1) [seeand]. 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

Indications & Related Conditions

Conditions associated via SNOMED clinical relationships

Administration & Protocol

Dosing, route, and treatment protocol

Detailed dosage and administration information is available in the full FDA drug label.

View full prescribing information on DailyMed

Monitoring & Follow-Up

Biomarkers relevant to this intervention via related conditions

No monitoring biomarkers have been mapped yet. Biomarker-intervention linkages are derived through related conditions and will expand as the knowledge graph grows.

Research Evidence

Published studies and systematic reviews

Sort:
Modeling the public health impact of voxelotor in the management of sickle cell disease in France.
Galacteros Frédéric, Ethgen Olivier, Beillat Maud · PLoS One · 2023
PMID: 37703228Meta-AnalysisFull text (PMC)
The efficacy of voxelotor, 900 mg in patients with sickle cell anaemia: A meta-analysis of the randomised controlled trials.
Tanriverdi Lokman Hekim, Sarici Ahmet, Erkurt Mehmet Ali et al. · Int J Clin Pract · 2021
PMID: 33369007Meta-Analysis
Systematic Review of Voxelotor: A First-in-Class Sickle Hemoglobin Polymerization Inhibitor for Management of Sickle Cell Disease.
Han Jin, Saraf Santosh L, Gordeuk Victor R · Pharmacotherapy · 2020
PMID: 32343424Meta-Analysis
Effect of voxelotor on cerebral perfusion and cerebral oxygen metabolism and cardiac stress in adult patients with sickle cell disease.
Konté Kadère, Afzali-Hashemi Liza, Baas Koen P A et al. · Am J Hematol · 2025
PMID: 39564863TrialFull text (PMC)
The influence of voxelotor on cerebral blood flow and oxygen extraction in pediatric sickle cell disease.
Brothers Rowan O, Turrentine Katherine B, Akbar Mariam et al. · Blood · 2024
PMID: 38364110TrialFull text (PMC)
Effect of voxelotor on cardiopulmonary testing in youths with sickle cell anemia in a pilot study.
Phan Vivian, Hershenson Jared, Caldarera Laura et al. · Pediatr Blood Cancer · 2023
PMID: 37247190Trial
Pharmacokinetics of Voxelotor in Patients With Renal and Hepatic Impairment.
Preston Richard A, Marbury Thomas, Balaratnam Ganesh et al. · J Clin Pharmacol · 2021
PMID: 33084052TrialFull text (PMC)
Pharmacokinetics and pharmacodynamics of voxelotor (GBT440) in healthy adults and patients with sickle cell disease.
Hutchaleelaha Athiwat, Patel Mira, Washington Carla et al. · Br J Clin Pharmacol · 2019
PMID: 30743314TrialFull text (PMC)
A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease.
Vichinsky Elliott, Hoppe Carolyn C, Ataga Kenneth I et al. · N Engl J Med · 2019
PMID: 31199090Trial

Research data from MEDLINE/PubMed

Benefits & Expected Outcomes

Benefits, expected outcomes, efficacy data, and NNT (Number Needed to Treat) are pending physician authorship and evidence review.

Risks & Side Effects

Adverse reaction and safety data for this drug is sourced from the FDA-approved label.

View adverse reactions & drug interactions on DailyMed

Related Symptoms

Symptoms associated with conditions this intervention addresses

No related symptoms have been mapped yet. Symptom linkages are derived through associated conditions.

Alternatives & Comparisons

Alternative treatments, comparison data, and clinical decision support are pending physician authorship.

Medical Disclaimer

This information is for educational purposes only and does not constitute medical advice, diagnosis, or treatment. Always consult with a qualified healthcare provider regarding any medical condition or treatment plan.

Do not start, stop, or change any treatment without consulting your healthcare provider.

Quick Facts

Type
Substance / Medication
Fully Specified Name
Voxelotor (substance)
SNOMED CT
833284006
UMLS CUI
C4723629
RxNorm CUI
2265678
Labeler
Global Blood Therapeutics, Inc, A subsidiary of Pfizer Inc.

Clinical Data

This intervention maps to 2 entities in the Ltrl knowledge graph.

2
Conditions
0
Biomarkers
0
Specialists
0
Symptoms

Data is sourced from SNOMED CT, UMLS, and the Ltrl clinical knowledge graph. Content sections marked as pending require physician authorship. Consult a healthcare provider before starting any treatment.

Voxelotor: Uses, Evidence & Side Effects | Ltrl