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
Impact of Voxelotor on Red Blood Cell Exchange Therapeutic Procedures: Evaluation of Multi-Institutional Procedure Data.
Campbell-Lee Sally, Jin Ming, Fortuny Lisandro et al. · J Clin Apher · 2025
PMID: 40391631ObservationalFull text (PMC)
Voxelotor as a Treatment of Persistent Hypoxia in the ICU.
Al-Qudsi Omar, Reynolds John M, Haney John C et al. · Chest · 2023
PMID: 37423700Case Report
The effect of Voxelotor on quantitation of HbS levels by high-performance liquid chromatography in a patient with sickle cell disease.
Giacomini Luca, Puricelli Chiara, Sacchetti Sara et al. · Int J Lab Hematol · 2023
PMID: 37604778Case Report
Acute venous thromboembolism after initiation of voxelotor for treatment of sickle cell disease.
Lemon Natalie, Sterk Ethan, Rech Megan A · Am J Emerg Med · 2022
PMID: 34991907Case Report
Urgent use of voxelotor in sickle cell disease when immediate transfusion is not safe.
Ferlis Mica, Lipato Thokozeni, Roseff Susan D et al. · Eur J Haematol · 2022
PMID: 35848827Case ReportFull text (PMC)

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 Healos knowledge graph.

2
Conditions
0
Biomarkers
0
Specialists
0
Symptoms

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