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Oxbryta (Voxelotor)

Pfizer Inc. · FDA-approved approved Small molecule Verified Quality 83/100

Hemoglobin S polymerization inhibitor that binds HbS 1:1 and increases oxygen affinity.

Voxelotor (Oxbryta) is a small molecule hemoglobin S polymerization inhibitor approved under accelerated approval for sickle cell disease in patients 4 years and older based on hemoglobin increase. The drug demonstrates linear pharmacokinetics with a 38.7-hour half-life, extensive hepatic metabolism via multiple CYP enzymes, and preferential distribution to red blood cells. Major risks include serious hypersensitivity reactions (contraindicated in prior hypersensitivity), significant CYP3A4 drug interactions requiring dose adjustments or avoidance, and laboratory test interference with hemoglobin subtype measurement. Continued approval is contingent on verification of clinical benefit in confirmatory trials.

At a glance

Generic nameVoxelotor
SponsorPfizer Inc.
Drug classHemoglobin S polymerization inhibitor
TargetHemoglobin S (HbS)
ModalitySmall molecule
Therapeutic areaHematology
PhaseFDA-approved
Annual revenue250

Mechanism of action

Voxelotor is a hemoglobin S polymerization inhibitor that binds to HbS with a 1:1 stoichiometry and exhibits preferential partitioning to red blood cells. By increasing the affinity of hemoglobin for oxygen, voxelotor demonstrates dose-dependent inhibition of HbS polymerization. Nonclinical studies suggest that voxelotor may inhibit RBC sickling, improve RBC deformability, and reduce whole blood viscosity.

Approved indications

Common side effects

Drug interactions

Patents

PatentExpiryType

Primary sources

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SourceUsed for
FDA Orange BookPatents + exclusivity
SEC EDGARRevenue + earnings

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