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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

  1. 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
  2. Brooks M FDA OKs Voxelotor (Oxbryta) for Root Cause of Sickle Cell Disease . Medscape - Nov 25, 2019. https://www.medscape.com/viewarticle/921815
  3. 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