Nova Laboratories’ Xromi (hydroxycarbamide) has received approval from the Medicines and Healthcare products Regulatory Agency and the European Commission to extend the indication for vaso-occlusive complications of sickle cell disease (SCD) to infants from nine months of age.
SCD is a group of inherited red blood cell disorders that are caused by a genetic mutation that affects the production of haemoglobin, the protein in red blood cells that carries oxygen.
The regulatory decisions were based on data from the pharmacokinetic, efficacy and safety study, HUPK, which showed that Xromi is expected to work in the same way in children from nine months of age as it does in older children.
Furthermore, additional data from the phase 3 BABY HUG study suggests that the benefits and safety of Xromi in children as young as nine months of age are similar to those in older children.
Dr James White, deputy managing director, Nova Laboratories, commented: “[The] approval to extend the indication to infants has the potential not only to alleviate the immediate symptoms but to significantly improve long-term outcomes and quality of life for those born with this genetic disorder.”
Baba Inusa, professor of paediatric haematology, Guy’s and St Thomas’ Hospital, said: “The results of the HUPK study, as well as a robust body of evidence from multiple studies conducted over the past decade, demonstrate Xromi’s efficacy in reducing complications associated with SCD, not just in adults and older children but also in infants.”
Xromi was previously approved in the UK and EU in 2019 for the prevention of vaso-occlusive complications of SCD in patients over the age of nine months and, most recently, by the Scottish Medicines Consortium in 2020 for children aged between two and nine years.
It is currently the only licensed liquid formulation of Xromi and offers a child-friendly alternative to tablets or capsules, which allows for accurate and precise dosing to be tailored to each child’s weight and condition by healthcare providers.
