Rilzabrutinib for treating persistent or chronic immune thrombocytopenia

Rilzabrutinib is currently in clinical development for the treatment of persistent or chronic immune thrombocytopenia (ITP) in adult. ITP is an auto-immune disease that arises when the body’s immune system attacks platelet cells resulting in a decreased number of platelets (platelet count) and impairs platelet production. Platelets are a specialised type of blood cell that help blood to clot following damage to a blood vessel wall so if there is a decreased platelet count there is an increased risk of frequent bleeding or severe bleeding. This results in the symptoms associated with ITP such as petechial (pin prick rash of blood spots), bruising, nosebleeds, gum bleeds, fatigue and heavy periods. Despite the range of available therapies, a need remains for safer therapy that results in durable increases in platelet count, eliminates related long-term toxic effects, and improves quality of life.