Voxelotor is a drug used to inhibit the polymerization of hemoglobin S, preventing the painful and sometimes lethal vaso-occlusive crises associated with sickle cell disease. This is a genetically inherited condition most prevalent in the Middle East, Africa, and certain parts of India.
- Mechanism
- Curator reviewed · 9 references
Sickle cell disease is characterized by deoxygenated sickle hemoglobin (HbS) polymerization. The genetic mutation causing this disease leads to the formation of abnormal, sickle-shaped red blood cells that aggregate and block blood vessels throughout the body, causing vaso-occlusive crises. Sickle-shaped red blood cells cannot effectively bind oxygen, thus incapable of allowing normal blood flow to organs.
Voxelotor increases Hb oxygen affinity. It binds reversibly to hemoglobin (Hb) by forming a covalent bond with the N‐terminal valine of the α‐chain of the protein, resulting in an allosteric modification of Hb. Voxelotor stabilizes the oxygenated Hb state and prevents HbS polymerization by increasing hemoglobin’s affinity for oxygen.
- Primary indication
- In the US, voxelotor is indicated to treat sickle cell disease in both adult and pediatric patients aged 4 years and older.Curator reviewed · 3 structured indications
- Formula / weight
- C19H19N3O3 · 337.379 g/mol (avg)
- First approval
- United States, 2019
- Code names
- GBT-440 · GTX-011
- Brand names
Resolves to
FWCVZAQENIZVMY-UHFFFAOYSA-NSMILESCC(C)N1N=CC=C1C1=C(COC2=CC=CC(O)=C2C=O)C=CC=N1What you can answer from here — as of June 19, 2026
Which drugs share a target with Voxelotor, and which of those have an active Phase 3 trial?
Create a free account or log in to explore the full data card.
Create Account