UK-based pharmaceutical manufacturer Nova Laboratories has unveiled details of its new liquid formulation of hydroxycarbamide at the Annual Sickle Cell and Thalassaemia conference in London.
Xromi, used to treat sickle cell disease, has been in development for four years. The product 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.
Solid dosage forms of the drug have been used in the treatment of sickle cell disease since the 1990s.
The liquid form of the treatment makes administering the drug to children as young as two-years-old significantly easier. It will also enable doctors to personalise doses for young children.
Nova Laboratories has invested in the region of £5.2m in product development and trials of the liquid form of the drug.
The company has also invested significantly in its UK manufacturing facility in the Midlands, in advance of the predicted strong demand for Xromi when it becomes available on prescription via the NHS later this year.
Around 15,000 people in the UK suffer from sickle cell disease. 20% of these are children under 16 years.
Dr Hussain Mulla, Head of Clinical Development at Nova Laboratories, said that 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.
“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.
“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.”
Dr Mulla added that 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”.
“We firmly believe that Xromi will significantly improve the lives of young patients suffering from the disease,” Dr Mulla concluded.
Xromi will be available on prescription only in the UK, EU and the Middle East through sickle cell disease healthcare specialists, but Nova says worldwide availability is planned.