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.
Ropeginterferon alfa-2b for injection is under development for the treatment of polycythaemia vera (PV), a rare blood disease in which the body makes too many red blood cells. The extra red blood cells make the blood thicker than normal and as a result, blood clots can form more easily. These clots may block blood flow through arteries and veins, which can cause a heart attack or stroke. Thicker blood also does not flow as quickly and may prevent organs from getting enough oxygen. A mutation, or change, in a gene called JAK2 is the major cause of PV. This gene makes a protein that helps the body produce blood cells. PV develops slowly and may not cause symptoms for years. PV has no cure, but treatments can help control the disease and its complications.