voxelotor (Oxbryta)
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Indications
- sickle cell disease (FDA-approved Nov 2019)[2]
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
More general terms
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: https://www.ncbi.nlm.nih.gov/pubmed/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: https://www.ncbi.nlm.nih.gov/pubmed/31199089 - ↑ 2.0 2.1 Brooks M FDA OKs Voxelotor (Oxbryta) for Root Cause of Sickle Cell Disease . Medscape - Nov 25, 2019. https://www.medscape.com/viewarticle/921815