Apadamtase alfa for treating acute thrombotic thrombocytopenic purpura (TTP) episodes associated with congenital TTP (cTTP) and long-term prophylaxis in patients with congenital ADAMTS-13 deficiency

Apadamtase alfa is currently in clinical development for treating thrombotic thrombocytopenic purpura (TTP) episodes associated with congenital TTP (cTTP) and long-term prophylaxis in patients with congenital ADAMTS-13 deficiency. Congenital ADAMTS-13 occurs when genetic mutations lead to low levels of the ADAMTS-13 enzyme (a protein). This condition is characterised by recurrent episodes of acute (sudden onset) TTP - a rare, serious blood disease, and sub-acute manifestations in the intervening periods. It typically presents either in infancy or later in life, particularly in pregnancy. Major manifestations may include a severe decrease in the number of blood platelets (thrombocytopenia), abnormal destruction of red blood cells (hemolytic anemia), and disturbances in the nervous system and other organs occur due to small clots that form in the smallest arteries. There is a need for treatments that directly increase ADAMTS-13 levels. Since apadamtase alfa replaces ADAMTS-13, it may prevent/control TTP flare-ups.