FDA grants priority review to Oxbryta for sickle cell disease
The FDA granted priority review to voxelotor for the treatment of children aged 4 to 11 years with sickle cell disease.
The agency also granted priority review to a new drug application that seeks approval for a new dispersible tablet dosage form of voxelotor (Oxbryta, Global Blood Therapeutics).
The FDA is expected to make decisions on both applications by Dec. 25.
Voxelotor directly targets hemoglobin polymerization, the root cause of red blood cell sickling in sickle cell disease.
The FDA previously granted accelerated approval to the agent in tablet dosage form to treat patients aged 12 years or older. An estimated 17,000 children in the United States aged 4 to 11 years have sickle cell disease.
“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 [our company’s] goal of bringing Oxbryta to all eligible patients suffering from this devastating disease,” Ted W. Love, MD, president and CEO of Global Blood Therapeutics, said in a company-issued press release. “There are few current therapeutic options for children under 12 years of age with sickle cell disease, which can cause irreversible multiorgan 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.”
The FDA based the priority reviews on results of the open-label, phase 2A HOPE-KIDS 1 study, which included 45 children aged 4 to 11 years with sickle cell disease.
Results showed weight-based treatment with the Oxbryta dispersible tablets dosage led to rapid and sustained improvements in hemoglobin, as well as concurrent reduction of hemolysis.