Liquid formulation for treatment of sickle cell now available

The EMA licensed liquid-form of hydroxycarbamide developed by pharmaceutical manufacturer Nova Laboratories is now available to sickle cell disease specialists in the UK and Ireland.

Set to revolutionise the control of sickle cell disease in children as young as two-years-old, the product Xromi is used to treat the disease and represents a significant evolution of solid dosage forms of the drug hydroxycarbamide that have been used in the treatment of sickle cell disease since the 1990s.

Xromi became available on prescription via the NHS on 23rd March 2020, and carries a two-year expiry date.

Granted a license by the European Medicines Agency (EMA) in July last year, and unveiled at the Annual Sickle Cell and Thalassaemia conference in London last October, Xromi is set to transform the control of sickle cell disease in young children because of its liquid form and strawberry flavour, which together make it easier for small children to take than the tablet form, as well as adolescents and adults with sickle cell disease who struggle to take tablets.

Dr Hussain Mulla, Head of Clinical Development at Nova Laboratories, explains: “Xromi is a new liquid formulation of hydroxycarbamide that makes administering the drug to children as young as two-years-old significantly easier and for the first time means doctors can personalise doses for young children.

“Nova Laboratories has invested £5.2m in Xromi’s intense four-year product development programme and trials of the liquid-form of the drug. We have also expanded our UK facility and global HQ in Leicestershire where Xromi is manufactured.”

He adds: “Xromi marks a new and major development in the treatment of sickle cell disease. We firmly believe that Xromi will significantly improve the lives of young patients suffering from the disease.”

Xromi is available on prescription-only to sick cell disease patients in the UK, EU and the Middle East through sickle cell disease healthcare specialists. Nova is planning for worldwide availability as soon as possible: “We have plans to reach the global market in order to help as many patients as possible to manage their symptoms and reduce vaso-occlusive complications,” says Dr Mulla.

Around 15,000 people in the UK suffer from sickle cell disease, 20 percent of whom are children under 16 years.

Hydroxycarbamide is a safe treatment modality and can boost foetal haemoglobin to levels that prevent red blood cells sickling and significantly reduce vaso-occlusive complications.

Dr Mulla adds: “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. Xromi is the first hydroxycarbamide formulation appropriate for young children, and as its liquid formula permits accuracy and precision, doctors can at last personalise doses in children.”

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