12-24-2021

U.S. FDA Approves Supplemental New Drug Application for Expanded Indication of Oxbryta® for Children as Young as 4 Years of Age with Sickle Cell Disease

 Global Blood Therapeutics , Inc. (GBT) (NASDAQ: GBT) today announced the U.S. Food and Drug Administration (FDA) has granted accelerated approval of a supplemental New Drug Application (sNDA) for Oxbryta® (voxelotor) tablets for the treatment of sickle cell disease (SCD) in children ages 4 to less than 12 years. This approval expands the previously approved use of Oxbryta to treat SCD in patients ages 12 years and older in the United States. The FDA also approved GBT’s separate New Drug Application (NDA) for Oxbryta (voxelotor) tablets for oral suspension, a new dispersible, once-daily tablet dosage form suitable for patients ages 4 to less than 12 years as well as for older patients who have difficulty swallowing whole tablets. Oxbryta is the first and only approved medicine that directly targets sickle hemoglobin polymerization, the root cause of the sickling and destruction of red blood cells in SCD.

“For decades, the sickle cell disease community has been profoundly underserved, and there have been limited treatment options for younger patients with their whole lives ahead of them. Complications of SCD that can cause irreversible organ damage are known to begin in the first few years of life, which is why earlier intervention is critical,” said Ted W. Love, M.D., president and chief executive officer of GBT. “Today’s FDA approval of Oxbryta for children as young as 4 years old – and with a pediatric-friendly dosage form – is an important advance in the treatment of this devastating, lifelong condition. GBT is proud to lead the development of new medicines to address the inadequacies of care for SCD patients.”

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