The Food and Drug Administration has accepted for Priority Review the supplemental New Drug Application (sNDA) for voxelotor (Oxbryta®) for the treatment of sickle cell disease in patients aged 4 to 11 years, along with the New Drug Application (NDA) for a new age-appropriate dispersible tablet dosage form of voxelotor for pediatric patients.

Voxelotor is a sickle hemoglobin polymerization inhibitor that binds to sickle hemoglobin and exhibits preferential partitioning to red blood cells. The sNDA and NDA are supported by data from the open-label phase 2a HOPE-KIDS1 study (ClinicalTrials.gov Identifier: NCT02850406), which evaluated the efficacy and safety of voxelotor 300mg dispersible tablets in 45 patients aged 4 to 11 years with sickle cell disease. 

Findings showed that weight-based treatment with voxelotor achieved rapid and sustained improvements in hemoglobin as well as concurrent reduction of hemolysis. The safety profile of voxelotor in patients 4 to 11 years of age was consistent with that observed in patients 12 years of age and older with no new safety signals identified.

A Prescription Drug User Fee Act target date of December 25, 2021 has been set for both applications. 


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“The FDA’s acceptance of our regulatory submissions for Oxbryta for the treatment of sickle cell disease in children ages 4 to 11 years and a pediatric-friendly dosage form of Oxbryta is an important step toward achieving GBT’s goal of bringing Oxbryta to all eligible patients suffering from this devastating disease,” said Ted W. Love, MD, president and CEO of GBT. “There are few current therapeutic options for children under 12 years of age with sickle cell disease, which can cause irreversible multi-organ damage in the first few years of life.” 

Voxelotor is currently marketed under the trade name Oxbryta and is indicated for the treatment of sickle cell disease in patients 12 years of age and older. The product is currently available as 500mg tablets.

Reference

US FDA accepts for Priority Review supplemental New Drug Application for Oxbryta® (voxelotor) for the treatment of sickle cell disease in children ages 4 to 11. News release. Global Blood Therapeutics, Inc. Accessed September 7, 2021. https://www.globenewswire.com/news-release/2021/09/07/2292458/37049/en/U-S-FDA-Accepts-for-Priority-Review-Supplemental-New-Drug-Application-for-Oxbryta-voxelotor-for-the-Treatment-of-Sickle-Cell-Disease-in-Children-Ages-4-to-11.html