The European Medicines Agency's (EMA's) Committee for Medicinal Products for Human Use recommended granting marketing authorization for hydroxycarbamide (Xromi, Nova Laboratories Ireland Limited) for the prevention of vaso-occlusive complications of sickle cell disease in patients older than 2 years.
Hydroxycarbamide is a ribonucleotide reductase inhibitor that interferes with the synthesis of DNA without disrupting the synthesis of ribonucleic acid or protein. Its benefits include the ability to reduce vaso-occlusive sickle cell disease complications.
Xromi is a hybrid medicine of Hydrea, a medication that has been authorized in the European Union since May 29, 1986. Although Xromi and Hydrea have the same active substance, Hydrea is authorized for the different indications of certain cancers. Applications for hybrid medicines depend in part on findings from preclinical tests and clinical trials of a reference product that is already authorized and partly on new data.
Hydroxycarbamide will be available as an oral solution containing 100 mg/mL.
The most frequently seen adverse effects are related to bone marrow depression and include "neutropenia, reticulocytopenia, macrocytosis, thrombocytopenia, anaemia, headache, dizziness, nausea, constipation, skin ulcer, oral, nail and skin hyperpigmentation, dry skin and alopecia," According to an EMA release.
The full indication for Xromi is "prevention of vaso-occlusive complications of sickle cell disease in patients over 2 years of age."
The committee proposes that Xromi be prescribed by physicians or other healthcare professionals with experience managing patients with sickle cell disease.
The summary of product characteristics will include detailed recommendations for use of the product and will be published in the European public assessment report, which will be made available in all official European Union languages after the European Commission grants marketing authorization.
Cite this: EU Panel Recommends Hydroxycarbamide for Sickle Cell Disease - Medscape - Apr 26, 2019.