GBT’s Oxbryta® Accepted by FDA for Priority Review for Pediatric Sickle Cell Disease

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. Findings showed that weight-based treatment with voxelotor achieved rapid and sustained improvements in hemoglobin as well as concurrent reduction of hemolysis.

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.

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. 

Global Blood Therapeutics (GBT) is a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities. Founded in 2011, GBT is delivering on its goal to transform the treatment and care of sickle cell disease (SCD), a lifelong, devastating inherited blood disorder.

GBT’s goal is to build on Oxbryta momentum with labeling and geographic expansions. GBT plans a thoughtful and sustainable approach to achieving worldwide access for Oxbryta over time. Oxbryta is available through two specialty pharmacies: Accredo and CVS Specialty.

Takeaway: GBT’s vision is to optimize patient options and increases the potential addressable market for Oxbryta