Voxelotor (previously known as GBT440) is a medication developed by Global Blood Therapeutics (GBT) for the treatment of sickle cell disease. It is an oral tablet that works by targeting hemoglobin, the oxygen-carrying molecule in red blood cells. Voxelotor prevents sickle cell disease by increasing the amount of oxygen in the blood, reducing the sickling of red blood cells, and improving the flow of blood through the body. Voxelotor is currently approved by the FDA and is the first medication to be approved for the treatment of sickle cell disease in more than 20 years. Clinical trials have shown that Voxelotor can improve hemoglobin levels, reduce red blood cell sickling, and reduce anemia symptoms in patients with sickle cell disease.
Voxelotor, sold under the brand name Oxbryta, is a medication used to treat sickle cell disease (SCD) in adults and children four years of age and older [1, 2]. SCD is an inherited blood disorder that affects red blood cells, causing them to become sickle-shaped and impede blood flow.
How Voxelotor Works:
- Voxelotor belongs to a class of medications called hemoglobin S (HbS) polymerization inhibitors [1].
- Hemoglobin is the protein in red blood cells that carries oxygen throughout the body. In SCD, an abnormal form of hemoglobin, called hemoglobin S, can cause red blood cells to sickle and clump together [3].
- Voxelotor works by stabilizing hemoglobin S and preventing it from polymerizing (clumping together) [2]. This helps red blood cells maintain their normal shape and allows them to flow more easily through blood vessels.
Benefits of Voxelotor:
- By preventing red blood cell sickling, Voxelotor can help to reduce the frequency and severity of pain crises, a hallmark symptom of SCD [2, 4].
- It may also help to decrease the need for blood transfusions and hospitalizations in people with SCD [4].
Dosage and Administration:
- Voxelotor comes as a tablet taken orally, once daily, with or without food [2, 4].
- A doctor will determine the appropriate dosage based on individual factors like age, weight, and overall health.
Important Considerations:
- Voxelotor is not a cure for SCD, but it can help to manage the symptoms and improve quality of life for people with the condition [4].
- Some common side effects of Voxelotor include headache, diarrhea, abdominal pain, and fatigue [2].
- It can also lower red blood cell counts, so regular blood monitoring is required while taking this medication [4].
- Voxelotor may interact with other medications, so it's crucial to inform your doctor about all medications, supplements, and herbal products you're taking [4].