Sickle Cell Oral Treatment Xromi Approved in Scotland for Ages 2–9

The Scottish Medicines Consortium (SMC) has approved the use of Xromi (hydroxycarbamide), a liquid form of hydroxyurea, for treating children with sickle cell disease (SCD).

Xromi is expected to be used in children older than 2 and younger than 9 who have difficulty swallowing tablets.

“This is great news for the sickle cell community in Scotland and shows the promising trajectory of treatments being made available for people living with sickle cell across the UK,” the U.K.-based Sickle Cell Society stated in a press release. 

The approval was based on a recent assessment by the SMC of a 100 mg/mL solution of Xromi. The SMC  advises the National Health Service Boards and the Area Drug and Therapeutic Committees Collaborative on therapies’ use in Scotland.

SCD is caused by an abnormal version of hemoglobin, the protein that carries oxygen in the blood. The abnormal hemoglobin is known as hemoglobin S, or sickle hemoglobin, because it leads to sickle-shaped red blood cells becoming stuck inside blood vessels and forming clumps. “Sickled” cells have short lives compared to healthy, round red blood cells, which can result in anemia.

Xromi is a strawberry-flavored oral formulation used to treat sickle cell in patients 2 and older to prevent a so-called vaso-occlusive crisis (VOC) — a painful complication that occurs when blood vessels become blocked by sickled red blood cells.

The therapy is intended to decrease the number and severity of VOC episodes by reducing hemoglobin S and some cells such as red blood cells. It also increases the levels of hemoglobin F, a more efficient type of hemoglobin found at high levels in the early stages of development. 

Patients usually are started on a daily dose of 15 mg/kg (milligrams per kilogram of weight) of Xromi, and increased, if necessary, every eight weeks to a maximum of 35 mg/kg. The medicine is supplied with two oral syringes — a small red syringe to deliver doses up to 3 mL, and a larger white syringe for higher volumes. Patients are advised to drink water after Xromi treatment to ensure the full dose reaches the stomach.

The Sickle Cell Society, along with manufacturers Nova Laboratories, said it worked to provide clear and easy-to-understand instructions on the use of Xromi, which is already available in England and Wales.