Tecarfarin is in clinical development for the prevention of thromboembolism in patients requiring long-term blood thinning treatment. Venous thromboembolism (VTE) refers to a blood clot that develops in a vein. There are two types of VTE: deep vein thrombosis (DVT) which refers to a blood clot in a vein and pulmonary embolism (PE) which refers to a blood clot that has broken free and travelled to the lungs.. Some patients who have had a VTE require long- term treatment with blood-thinning medication to reduce the risk of VTE recurrence.
Tecarfarin, a vitamin K reductase, is given by oral administration and works by blocking the liver from using vitamin K to make clotting factors. Clotting factors work with blood cells called platelets that trigger the clotting process to form a blood clot. Stopping the activation of these vitamin K dependent clotting factors means the blood takes much longer to clot so there is a decreased risk of VTE. Tecarfarin works in the same way as warfarin, which is the current best treatment option for preventing VTE, but is expected to be safer and deliver more predictable clotting. If licensed, tecarfarin would offer an additional treatment option for preventing VTE in patients who require long-term anticoagulation therapy.
Luspatercept is under clinical development for the treatment of adult patients with beta-thalassaemia who don’t regularly require blood transfusion. Thalassaemia is a commonly inherited blood disorder resulting from an abnormality in one of the genes that affects the production of haemoglobin, a protein in red blood cells that carries oxygen throughout the body . Beta-thalassaemia is a subtype caused by a specific gene mutation. People with thalassaemia produce either little or no normal haemoglobin. Current treatment options for beta-thalassaemia are limited to blood transfusions with its associated risks and complications.