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® (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.”
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