Is Voxelotor approved by the FDA?

Yes, voxelotor, marketed under the brand name Oxbryta, is FDA approved. The U.S. Food and Drug Administration (FDA) approved voxelotor on November 25, 2019, for the treatment of sickle cell disease in adults and children aged 12 years and older. The approval was expanded in December 2021 to include children as young as 4 years old.

What is Voxelotor?

Voxelotor is a medication used to treat sickle cell disease, a genetic disorder that causes red blood cells to become misshapen and break down. Voxelotor works by inhibiting hemoglobin polymerization, which is a key factor in the pathophysiology of sickle cell disease. This action helps reduce hemolysis (breakdown of red blood cells) and improve anemia.

Dosage and Administration

Voxelotor is available in oral tablet forms and is usually taken once daily. The dosage varies depending on the patient's age and weight:

• Adults and Children 12 years and older: 1500 mg orally once a day.
• Children 4 to less than 12 years old:
  • Weight: 10 to less than 20 kg: 600 mg orally once a day.
  • Weight: 20 to less than 40 kg: 900 mg orally once a day.
  • Weight: 40 kg or greater: 1500 mg orally once a day.

The medication can be taken with or without food. It is essential to follow the prescribed dosing instructions and not to crush, chew, or break the tablets. The tablets for oral suspension should be mixed with room temperature clear liquid just before taking.

Side Effects

Common Side Effects

• Headache
• Fever
• Hives, rash
• Stomach pain
• Nausea
• Diarrhea
• Vomiting

Serious Side Effects

• Signs of allergic reactions such as hives, difficulty breathing, and swelling of the face, lips, tongue, or throat require emergency medical help.

Warnings and Precautions

• Allergic Reactions: Patients should be aware of signs of allergic reactions and seek immediate medical help if they occur.
• Liver Problems: Inform your doctor if you have a history of liver problems, as voxelotor can affect liver function.
• Pregnancy and Breastfeeding: The effects of voxelotor on an unborn baby are not fully known. Pregnant women or those planning to become pregnant should consult their doctor. Breastfeeding is not recommended during treatment and for at least 2 weeks after the last dose.

Drug Interactions

Voxelotor can interact with several medications, including:

• Phenobarbital and other barbiturates
• St. John's wort
• Antibiotics such as rifabutin, rifampin, and rifapentine
• Cancer medications like apalutamide and enzalutamide
• HIV medications including efavirenz and nevirapine
• Seizure medications like carbamazepine, oxcarbazepine, phenytoin, and primidone
• Steroid medications such as dexamethasone and prednisone

It is crucial to inform your healthcare provider about all medications you are taking to avoid potential interactions.

Conclusion

Voxelotor (Oxbryta) is an FDA-approved medication for the treatment of sickle cell disease in patients aged 4 years and older. Approved on November 25, 2019, it offers a significant therapeutic option to manage this chronic and often debilitating condition. As with any medication, it is essential to follow the prescribed dosage, be aware of potential side effects, and communicate with your healthcare provider about any other medications you are taking.

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