Waskyra

Waskyra is a medicine for treating people aged 6 months and older with Wiskott\-Aldrich syndrome (WAS) who have a mutation in the WAS gene. It is used to treat patients for whom a haematopoietic stem cell transplantation is appropriate, but for whom no suitable stem cell donor is available. Wiskott\-Aldrich syndrome is caused by abnormalities in the gene that produces the WAS protein, which is found in blood cells and certain cells of the immune system (the body’s natural defences). People with Wiskott\-Aldrich syndrome bruise and bleed easily because they have too few normal platelets (components that help the blood to clot); they also have frequent infections because they have too few normal immune cells, which could lead to sepsis (when bacteria and their toxins circulate in the blood and start damaging the organs). In addition, there is a higher risk of developing some types of cancer, such as lymphoma. Wiskott\-Aldrich syndrome is rare, and Waskyra was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 6 June 2012\. Waskyra contains the active substance etuvetidigene autotemcel, which consists of genetically modified stem cells taken from the patient’s own blood.