Benzinga | Sep 7, 2021 08:10AM EDT

Global Blood Therapeutics Announces FDA Has Accepted For Priority Review Supplemental New Drug Application For Oxbryta

New Drug Application for Oxbryta dispersible tablets also accepted for priority review

Approximately 17,000 children ages 4 to 11 years have sickle cell disease in the United States

SOUTH SAN FRANCISCO, Sept. 07, 2021 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT) today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing and review the company's supplemental New Drug Application (sNDA) seeking accelerated approval for Oxbryta(r) (voxelotor) for the treatment of sickle cell disease (SCD) in children ages 4 to 11 years and its New Drug Application (NDA) seeking approval for a new age-appropriate dispersible tablet dosage form of Oxbryta suitable for pediatric patients. Oxbryta, a first-in-class therapy that directly targets hemoglobin polymerization, the root cause of red blood cell sickling in SCD, is currently approved by the FDA in a tablet dosage form to treat SCD in patients age 12 years and older.

The FDA granted Priority Review for both the NDA and sNDA, providing a six-month review, and assigned a Prescription Drug User Fee Act (PDUFA) target action date for both applications of December 25, 2021.

"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, M.D., 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. Given the profound unmet need, we appreciate the FDA prioritizing the review of potential treatments for the long-overlooked sickle cell disease community."

In the U.S., approximately 17,000 children ages 4 to 11 years have sickle cell disease.1 The Oxbryta pediatric sNDA and NDA are based on data from the open-label Phase 2a HOPE-KIDS 1 Study (GBT440-007). An analysis of data presented at the European Hematology Association (EHA) 2021 Virtual Congress in 45 children with SCD ages 4 to 11 years enrolled in the study showed that weight-based treatment with the Oxbryta dispersible tablets dosage form that is the subject of the NDA resulted in rapid and sustained improvements in hemoglobin. Concurrent reduction of hemolysis (or red blood cell destruction) was also demonstrated.2

The NDA seeks approval for 300 mg dispersible tablets. The dispersible tablet formulation includes grape flavoring, is intended to be dispersed in room-temperature drinking water or other clear drinks for ease of swallowing, and allows for weight-based dosing in pediatric patients ages 4 to 11 years with SCD.

Priority review is granted to therapies that the FDA determines have the potential to provide a significant improvement in the safety or effectiveness of the treatment, diagnosis or prevention of a serious condition. Under the PDUFA, a priority review targets a review time of six months compared to a standard review time of 10 months.