Adzynma

Adzynma is a medicine used to treat children and adults with congenital thrombotic thrombocytopenic purpura (cTTP), an inherited disease caused by mutations (changes) in the ADAMTS13 gene. Patients with this disease have acute episodes in which thrombosis (blood clots) are formed in small blood vessels throughout their body. The clots can hamper the blood flow to the organs and cause damage. The increased clotting results in a shortage of platelets in the blood (thrombocytopenia), increasing the risk of bleeds. Patients also have small bleedings under the skin that appear as purple spots (purpura). cTTP also causes the body to break down red blood cells faster than the body can make them, lowering the level of red blood cells (microangiopathic haemolytic anaemia), with symptoms including fatigue, weakness and shortness of breath. cTTP is rare and Adzynma was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 3 December 2008. Further information on the orphan designation can be found on [EU/3/08/588 - orphan designation for treatment of thrombotic thrombocytopenic purpura](https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-588). Adzynma contains the active substance rADAMTS13.