Plasminogen (human) as intravenous infusion is in clinical development for people with hypoplasminogenemia. Hypoplasminogenemia is an ultra-rare, chronic, genetic condition associated with inflamed growths on the mucous membranes, the moist tissues that line body openings such as the eye, mouth, nasopharynx, trachea, and female genital tract. The growths are caused by the deposition of fibrin (a protein involved in blood clotting) and by inflammation. Growths can lead to severe medical problems including vision loss, ulcers of the gastrointestinal tract, and breathing difficulties caused by obstruction of the airway.
The medicinal product contains purified plasminogen extracted from human blood. Plasminogen is a naturally occurring protein that is primarily synthesized by the liver and circulates in the blood. Activated plasminogen, plasmin, is a fundamental component of the fibrinolytic system and is the main enzyme involved in the breaking down of blood clots and clearance of excess fibrin. Plasminogen is therefore vital in wound healing, cell migration, tissue remodelling, the formation of new blood vessels, and the development of embryos. When administered to patients who lack working plasminogen of their own, this plasma-derived protein replacement therapy is expected to replace the missing protein in their blood and correct the symptoms of the condition. If licensed, it may offer the first commercially available treatment option for patients with hypoplasminogenemia.
Valoctocogene roxaparvovec is a gene therapy; it can modify the genes (functional units of heredity) of individuals with haemophilia A so that they can produce the clotting protein needed to allow the blood to clot. If licensed, valoctocogene roxaparvovec would be the first gene therapy for severe haemophilia A. Valoctocogene roxaparvovec administered as a single treatment would be sufficient to maintain normal levels of factor VIII in adult males with severe haemophilia A, and might reduce the need for regular factor VIII prophylaxis (preventative treatment).