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voxelotor (Oxbryta)
Indications:
- sickle cell disease (FDA-approved Nov 2019) [2]
- voxelotor pulled from global markets, benefits no longer outweigh risks [3]
Dosage:
- 900 or 1500 mg QD
Adverse effects:
- headache & diarrhea are the most common adverse events
- abdominal pain, nausea, fatigue, rash, pyrexia
Mechanism of action:
- prevents polymerization of sickle hemoglobin upon deoxygenation
- increases blood hemoglobin in sickle cell disease
- diminishes hemolysis in sickle cell disease
General
hematologic agent
Database Correlations
PUBCHEM cid=71602803
References
- Vichinsky E, Hoppe CC, Ataga KI et al.
A phase 3 randomized trial of voxelotor in sickle cell disease.
N Engl J Med 2019 Jun 14;
PMID: 31199090
- Thompson A.
A targeted agent for sickle cell disease - Changing the protein but
not the gene.
N Engl J Med 2019 Jun 14;
PMID: 31199089
- Brooks M
FDA OKs Voxelotor (Oxbryta) for Root Cause of Sickle Cell Disease .
Medscape - Nov 25, 2019.
https://www.medscape.com/viewarticle/921815
- Bassett M
Sickle Cell Disease Drug Pulled From Global Markets.
Benefits of voxelotor (Oxbryta) no longer outweigh the risks, Pfizer says.
MedPage Today September 26, 2024
https://www.medpagetoday.com/publichealthpolicy/productalert/112123