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Novel treatment to target abnormality in sickle cell disease: Oxbryta approved by FDA


The FDA ( U.S. Food and Drug Administration ) has granted accelerated approval to Oxbryta ( Voxelotor ) for the treatment of sickle cell disease ( SCD ) in adults and pediatric patients 12 years of age and older.

Sickle cell disease is a lifelong, inherited blood disorder in which red blood cells are abnormally shaped, which restricts the flow in blood vessels and limits oxygen delivery to the body’s tissues, leading to severe pain and organ damage. It is also characterized by severe and chronic inflammation that worsens vaso-occlusive crises during which patients experience episodes of extreme pain and organ damage.
100,000 people in the U.S., and the more than 20 million globally, live with this debilitating blood disorder.

Oxbryta is an inhibitor of deoxygenated sickle hemoglobin polymerization, which is the central abnormality in sickle cell disease.
With Oxbryta, sickle cells are less likely to bind together and form the sickle shape, which can cause low hemoglobin levels due to red blood cell destruction.

Oxbryta’s approval was based on the results of a clinical trial with 274 patients with sickle cell disease.
In the study, 90 patients received 1500 mg of Oxbryta, 92 patients received 900 mg of Oxbryta and 92 patients received a placebo.
Effectiveness was based on an increase in hemoglobin response rate in patients who received 1500 mg of Oxbryta, which was 51.1% for these patients compared to 6.5% in the placebo group.

Common side effects for patients taking Oxbryta were headache, diarrhea, abdominal pain, nausea, fatigue, rash and pyrexia.

Oxbryta was granted Accelerated Approval, which enables the FDA to approve drugs for serious conditions to fill an unmet medical need based on a result that is reasonably likely to predict a clinical benefit to patients.
Oxbryta has also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. ( Xagena )

Source: FDA, 2019

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