In April 2019, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Xromi. It is a medicine used in adults, adolescents and children over two years of age who have sickle cell disease, a genetic disease where the red blood cells become rigid and sticky, and change from being disc-shaped to being crescent-shaped (like a sickle). Xromi is used to prevent so-called vaso-occlusive complications – problems that happen when blood vessels become blocked by the abnormal red blood cells, restricting the flow of blood to parts of the body. The applicant for this medicinal product is Nova Laboratories Ireland Limited. The active substance in Xromi, hydroxycarbamide, blocks the growth and reproduction of some cells, such as blood cells. Although the way that it works in this disease is not fully understood, hydroxycarbamide can reduce the numbers of cells that are circulating in the blood, as well as prevent red blood cells changing shape in patients with sickle cell disease.
Because hydroxycarbamide is a well-established substance that has been used for many years in the EU the company provided information from the published literature on the benefits and risks of hydroxycarbamide in its approved uses. This included information from 4 main studies in sickle cell disease, including 3 studies involving 384 adults and children in whom hydroxycarbamide was shown to substantially reduce the risk of severe, painful blockages of blood supply (called vaso-occlusive crises) compared with a dummy treatment, and a fourth study involving 121 children that showed hydroxycarbamide to be at least as effective as standard treatment using blood transfusions in reducing blood vessel damage in the brain and the risk of stroke. It also carried out a study that showed Xromi is bioequivalent to the reference medicine, Hydrea. Two medicines are bioequivalent when they produce the same levels of the active substance in the body.
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