New Form of Oxbryta Suitable for Children Now Available in US

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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A new formulation of Oxbryta (voxelotor) that is suitable for treating children as young as 4 is now available in the U.S. at specialty pharmacies.

Late last year, the U.S. Food and Drug Administration (FDA) expanded the approval of Oxbryta to treat children with sickle cell disease (SCD) as young as 4 — the therapy had previously been available as 500 milligram (mg) tablets and approved for use in patients ages 12 and older. At the time, the FDA also cleared a new oral suspension formulation of the therapy that is now available.

The 300 mg oral suspension tablets, available in bottles of 60 or 90, include grape flavoring and are designed to be dispersed in room-temperature drinks like water or clear soda.

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According to the therapy’s manufacturer, Global Blood Therapeutics (GBT), this method of administration may allow the therapy to be taken more easily by people who have difficulties swallowing whole tablets, especially younger children.

“The FDA’s recent approval of the new dispersible tablet dosage form of Oxbryta is an important advance for people and families affected by this devastating disease, and we are pleased to make this new dosage form available to patients by prescription,” Kim Smith-Whitley, MD, executive vice president and head of research and development at GBT, said in a press release.

“The sickle cell disease community needs more innovative, safe and effective treatment options for this disease,” Smith-Whitley said.

SCD is caused by mutations that lead to the production of an abnormal version of hemoglobin — the protein that red blood cells use to carry oxygen through the bloodstream. In SCD, faulty hemoglobin forms clumps, causing red blood cells to take on the irregular sicklelike shape that gives the disease its name.

Oxbryta is a small molecule that binds to hemoglobin and increases its ability to bind to oxygen. This helps prevent hemoglobin clumping and ultimately red blood cell sickling. According to GBT, Oxbryta is the first and only approved medication that addresses the root cause of SCD by directly targeting hemoglobin clumping.

GBT is also offering a support program called GBT Source Solutions that offers educational and financial support to patients who are prescribed Oxbryta.