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Leicestershire pharma manufacturer unveils revolutionary liquid form of sickle cell treatment

Leicestershire pharmaceutical manufacturer Nova Laboratories has unveiled details of its new liquid formulation of hydroxycarbamide at the Annual Sickle Cell and Thalassaemia conference held in London on 21 to 23 October 2019.

The product Xromi, used to treat sickle cell disease, 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.

Xromi, represents a significant evolution of the pill form of the drug that has been used in the treatment of sickle cell disease since the 1990s.

It makes administering the drug to children as young as two-years-old significantly easier and will enable doctors to personalise doses for young children.

In development for four years, Nova Laboratories has invested in the region of £5.2m in product development and trials of the liquid-form of the drug.

Nova, which employs around 250 staff, has also invested significantly in its manufacturing facility in Wigston, to manage the predicted strong demand for Xromi when it becomes available on prescription via the NHS later this year.

Sickle cell disease is a term covering a number of inherited health conditions that affect red blood cells. People with sickle cell disease produce unusually shaped red blood cells that can cause problems because they do not live as long as healthy blood cells and can block blood vessels. It is a serious and lifelong health condition, but treatment can help manage many of the symptoms.

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

“Hydroxycarbamide prevents red blood cells sickling and significantly reduces the frequency of pain crises and the need for blood transfusions in patients,” explains Dr Hussain Mulla, Head of Clinical Development at Nova Laboratories. “The current pill-form 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.

“Getting the dosing of the pill-form of the drug exactly right for such small children can be difficult,” says Dr Mulla.

“Xromi is the first formulation appropriate for young children, and its liquid formula will enable doctors to personalise doses in children, as it permits accuracy and precision. It has a pleasant strawberry flavour to aid palatability, and its liquid form will also benefit adolescents and adults with sickle cell disease who struggle to take tablets.”

Dr Mulla adds:

“The marketing of Xromi follows four years of intense drug development and 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 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:

“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.

For more information, visit: https://www.novalabs.co.uk/

 


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