FDA approves Oxbryta to treat sickle cell disease in young children

The US Food and Drug Administration has granted accelerated approval for Oxbryta (voxelotor), from USA-based Global Blood Therapeutics (Nasdaq: GBT), to treat sickle cell disease in pediatric patients aged four up to 11 years.

Oxbryta also on Friday was recommended for approval by the European Medicines Agency advisory panel. GBT's shares gained 5% to $29.03 in morning trading that day.

This approval expands the previously approved use of Oxbryta to treat SCD in patients ages 12 years and older in the USA. The FDA also approved GBT’s separate New Drug Application (NDA) for Oxbryta tablets for oral suspension, a new dispersible, once-daily tablet dosage form suitable for patients ages four 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.