ITP - idiopathic immune thrombocytopenic purpura

From Yenra

Chronic ITP is a serious autoimmune disorder characterised by low platelet counts in the blood (thrombocytopenia), which can lead to serious bleeding events. ITP is recognised as an orphan disease by the European Medicines Agency (EMEA) and there are an estimated 50,000 adult patients with chronic ITP in the EU. Nplate is a breakthrough thrombopoiesis-stimulating peptibody that represents a new approach to the management of ITP by increasing platelet production and potentially reducing the need for rescue and other maintenance medications.

In patients with ITP, platelets - or blood elements needed to prevent bleeding - are destroyed by the patient's own immune system. Low platelet counts leave adult ITP patients open to sudden serious bleeding events. The risk for serious bleeding events increases when platelet counts drop to less than 30,000 platelets per microliter; normal counts range from 150,000 to 400,000 platelets per microliter. ITP has historically been considered a disease of platelet destruction although recent data suggest that the body's natural platelet production processes in ITP are unable to compensate for low levels of platelets in the blood. Increasing the rate of platelet production may address low platelet levels associated with ITP.

Currently available treatments (i.e., corticosteroids, immunoglobulins) have limited application due to poor tolerability or transient effects. Surgical therapy (removal of the spleen) is also available to adult patients with chronic ITP, but does not work in all cases. Currently, there are 140,000 treated chronic ITP patients in Europe and the U.S. ITP affects about twice as many adult women as men.

Nplate romiplostim platelet producer is the first treatment specifically developed for ITP.