Bio Products Laboratory gets European approval for bleeding disorder treatment
UK-based Bio Products Laboratory has received marketing authorisation from the European Medicines Agency for Coagadex – a drug which treats a rare bleeding disorder.
The decision follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) in January.
Coagadex (human coagulation Factor X) is the first treatment to be specifically approved in Europe for the treatment of patients with hereditary factor X deficiency, and can be used preventatively or to treat bleeding episodes. It was granted orphan drug status in 2007.
Factor X deficiency is a lack of the Factor X protein in the blood. This protein plays a crucial role in blood clotting, which helps patients stop bleeding. People with Factor X deficiency are at increased risk of bleeding or experience excessive or prolonged bleeding.
In severe cases, usually in children, the condition can cause bleeding in the brain, lungs or gastrointestinal tract, which can be life-threatening. Hereditary Factor X affects only about 700 patients in Europe.
Coagadex was approved based upon data from two open-label, multicentre, prospective studies, where investigators found it to be effective at controlling bleeding episodes both during and after surgery.
Professor Flora Peyvandi, director of Angelo Bianchi Bonomi Hemophilia and Thrombosis Centre, hailed the approval of a specific treatment for the condition. She comments: “Until now, we have focused on treating factor X deficiency with blood infusions of plasma or a concentrate of clotting factors. We have wanted a specific factor X therapy to treat these vulnerable patients. Therefore, the approval of Coagadex in the EU today is a significant advance for patients.”