Drug information of voxelotor


Drug group:

Voxelotor is used to treat sickle cell disease in adults and children at least 12 years old.

Voxelotor is sometimes given together with another medicine called hydroxyurea.

Voxelotor was approved by the US Food and Drug Administration (FDA) on an "accelerated" basis. In clinical studies, some people responded to voxelotor, but further studies are needed.

Mechanism of effect

Voxelotor is an HbS polymerization inhibitor that reversibly binds to Hb and stabilizes the oxygenated Hb state. Through the increased Hb affinity for oxygen, voxelotor demonstrates dose-dependent inhibition of HbS polymerization, and may inhibit RBC sickling, improve RBC deformability, and reduce whole blood viscosity. Voxelotor may also extend RBC half-life and reduce anemia and hemolysis .



A high-fat, high-calorie meal increased voxelotor whole blood AUC and Cmax by 42% and 45%, respectively (compared to a fasted state); likewise, plasma AUC and Cmax increased by 42% and 95%, respectively.


Vd: Central compartment: 338 L; Peripheral compartment: 72.2 L; distributed predominantly into RBCs.


Primarily hepatic through phase I metabolism (oxidation and reduction), phase II metabolism (glucuronidation), and combinations of phase I and II metabolism. Oxidation is primarily by CYP3A4, with minor contribution from CYP2C19, CYP2B6, and CYP2C9.


Feces: ~63% (33% as unchanged drug); Urine: ~36% (0.08% as unchanged drug).

Clearance: 6.7 L/hour.

Time to Peak

Median: 2 hours.

Half-Life Elimination

35.5 hours.

Protein Binding


Drug indications

Sickle cell disease: Treatment of sickle cell disease in adults and pediatric patients ≥12 years of age.


Usual Adult Dose for Sickle Cell Anemia
1500 mg orally once a day

Drug contraindications

Serious hypersensitivity (eg, generalized rash, urticaria, mild shortness of breath, mild facial swelling, eosinophilia) to voxelotor or any component of the formulation.

Side effects

-This drug may be given with or without hydroxyurea.

Use: For the treatment of sickle cell disease.Usual Pediatric Dose for Sickle Cell Anemia12 years or older: 1500 mg orally once a day

-This drug may be given with or without hydroxyurea.

Use: For the treatment of sickle cell disease in patients 12 years or older.Side effects requiring immediate medical attentionAlong with its needed effects, voxelotor may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.Check with your doctor immediately if any of the following side effects occur while taking voxelotor:Rare
  • Black, tarry stools
  • chest pain
  • chills
  • cough
  • fever
  • hives or welts, itching or skin rash
  • painful or difficult urination
  • redness of the skin
  • sore throat
  • sores, ulcers, or white spots on the lips or in the mouth
  • swelling of the face
  • swollen glands
  • trouble breathing
  • unusual bleeding or bruising
  • unusual tiredness or weakness
Side effects not requiring immediate medical attentionSome side effects of voxelotor may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects.Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:More common
  • Diarrhea
  • headache
  • nausea
  • stomach pain



Points of recommendation

You should not use voxelotor if you are allergic to it.

Tell your doctor if you have ever had:

It is not known whether voxelotor will harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant.

Do not breastfeed while using this medicine, and for at least 2 weeks after your last dose.

Voxelotor is not approved for use by anyone younger than 12 years old.

Follow all directions on your prescription label and read all medication guides or instruction sheets. Use the medicine exactly as directed.

You may take voxelotor with or without food.

Swallow the tablet whole and do not crush, chew, or break it.

If you are receiving exchange transfusions, voxelotor can affect the results of certain blood tests. Tell any doctor who treats you that you are using voxelotor.

You should not stop using voxelotor without your doctor's advice.

Store at room temperature away from moisture and heat. Keep the tablets in their original container, along with the packet or canister of moisture-absorbing preservative.

Skip the missed dose and use your next dose at the regular time. Do not use two doses at one time.


Store at ≤30°C (86°F).

Pregnancy level


Benefit should outweigh risk

US FDA pregnancy category: Not assigned

Breast feeding warning

Not Recommended

Excreted into human milk: Unknown
Excreted into animal milk: Yes

-Because of the potential for changes in the hematopoietic system in the breastfed infant, breastfeeding is not recommended during treatment and for at least 2 weeks after the last dose.

There are no data on the effects of this drug on the breastfed infant or its effects on milk production. This drug is present in the milk of lactating rats.

Drug forms


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