‘Revolutionary’ liquid sickle cell treatment has been unveiled

Posted: 28 October 2019 | Rachael Harper (European Pharmaceutical Review) | No comments yet

A new liquid formulation of hydroxycarbamide to treat sickle cell disease, has been developed PharmaField has reported.  

The treatment, Xromi, was granted a license by the European Medicines Agency (EMA) in July 2019 and is set to “revolutionise the control of the disease in young children” the report said.

Nova Laboratories unveiled details of its new treatment formulation, saying that it makes administering the drug to children as young as two-years-old easier and enables doctors to personalise doses for young children.

Hydroxycarbamide is a safe treatment modality which can boost foetal haemoglobin to levels that prevent red blood cells sickling and significantly reduce vaso-occlusive complications, Dr Hussain Mulla, Head of Clinical Development at Nova Laboratories said in the report.

“The current pill form of hydroxycarbamide has been used in the treatment of sickle cell disease for more than 30 years. However, many children are not administered hydroxycarbamide until they develop serious acute or chronic clinical complications,” Dr Mulla continued. “The British Society for Haematology national treatment guidelines state that hydroxycarbamide should be started early in children, as a preventative medicine, since it can prevent long term complications of the disease and this was the driving force behind our decision to develop a formula suitable for young children.”

Getting the dosing of the pill form of the drug exactly right for very small children can be difficult and that the liquid form of the drug will “enable doctors to personalise doses in children, as it permits accuracy and precision,” Dr Mulla added.

Xromi will be available on prescription only in the UK, EU and the Middle East through sickle cell disease healthcare specialists, but Nova has reportedly said worldwide availability is planned.