New liquid formulation available for sickle cell disease

A new liquid formulation to treat sickle cell disease is now available in the UK and Ireland after being approved by the European Medicines Agency (EMA).

The liquid-form of hydroxycarbamide, developed by UK pharmaceutical manufacturer Nova Laboratories, will be available to sickle cell disease specialists in the UK and Ireland to treat children as young as two years old.

The product, Xromi, has been designed to make it easier to treat children with sickle cell disease through its liquid form, rather than the typical solid dosage forms of the drug which have been used since the 1990s.

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

Dr Hussain Mulla, head of Clinical Development at Nova Laboratories, said: “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 in the Midlands where Xromi is manufactured.

“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 a prescription-only basis to healthcare specialists in the UK, EU and the Middle East, with Nova planning for worldwide availability as soon as possible.

Dr Mulla added: “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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