Zynteglo is a medicine used to treat a blood disorder known as beta thalassaemia in patients 12 years and older who require regular blood transfusions. People with this genetic condition cannot make enough beta-globin, a component of haemoglobin, the protein in red blood cells that carries oxygen around the body. As a result, these patients have low red blood cell levels and need frequent blood transfusions. Zynteglo is used in patients who do not completely lack beta-globin and who are eligible for stem cell transplantation but do not have a matching related donor. Beta thalassaemia is rare, and Zynteglo was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 24 January 2013. Zynteglo contains as its active substance stem cells taken from the patients that have been genetically modified to contain a working gene for beta-globin. For more information click here.
Harmonizing care for rare diseases