The first and only treatment licensed specifically for the rare bleeding disorder hereditary factor X deficiency has now been launched across the UK.
Bio Products Laboratory's Coagadex was approved in Europe in March for the treatment and prophylaxis of bleeding episodes and for perioperative management in patients with the condition.
"Our previous focus has been on treating Factor X deficiency with blood infusions of plasma or a concentrate of clotting factors," noted Dr Steve Austin, Haemophilia Centre director of St George's University Hospitals NHS Foundation Trust, London, welcoming the therapy's launch. "For the first time, patients with this rare bleeding disorder can receive a specific factor X replacement that has been proven safe and effective in clinical studies."
Hereditary factor X deficiency affects around 700 patients in Europe, and is characterised by a lack of factor X protein in the blood, which plays a crucial role in coagulation.
People with this deficiency are at increased risk of bleeding, including inside the brain, lungs or gastrointestinal tract in the most severe cases, which can be life-threatening.
The UK is the first country in Europe where Coagadex will be made available, via a Commercial Medicines Unit tender, which starts on July 1st.
The CMU works on behalf of the Department of Health and the NHS, and in partnership with the people who buy pharmaceuticals for hospitals across the NHS in the UK.