U.S. FDA to review use of GBT’s blood disorder therapy in children

The US Food and Drug Administration accorded priority evaluation to two applications relating to Global Blood Therapeutics Inc’s therapy for sickle cell disease in children on Tuesday.

The business has applied for expedited approval of Oxbryta to treat the illness in children aged four to eleven years old, as well as permission for a dispersible tablet version of the medication.

The FDA has set a target action date of Dec. 25 for both applications, the company said.

Oxbryta was approved in the United States in 2019 in a tablet form to treat the disease in patients who are 12 years and older.

Sickle cell disease, which affects about 100,000 Americans, is an inherited blood disorder in which red blood cells have a crescent shape and can restrict flow in blood vessels.

The disorder is more prevalent in African-Americans and other minority groups, occurring in about one out of every 365 Black or African-American births.

Other treatments for children with the disease that have been approved in the United States include Siklos, a tablet developed by French drugmaker ADDMEDICA, and Emmaus Medical Inc’s oral powder Endari.

“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,” Global Blood Chief Executive Officer Ted Love said in a statement.

The company has also sought approval for Oxbryta in Europe to treat the disease in patients who are 12 years and older.