The government approved Takeda’s human protein C to treat severe congenital protein C deficiency, an extremely rare disease with only 13 Korean cases reported so far.

On Tuesday, the Ministry of Food and Drug Safety authorized the use of Ceprotin (ingredient: human protein C) to prevent and treat venous thrombosis and purpura fulminans in patients with severe congenital protein C deficiency.

Protein C deficiency is a type of blood clotting disease prone to thrombosis. The world’s first case was reported in 1982. Severe congenital protein C deficiency occurs in about one in 4 million newborns, and Korea has reported 13 patients so far.

In healthy people, the average protein C level is about 100 ng/dL, but in patients with severe congenital protein C deficiency, the level is less than 1 ng/dL. This disturbs the balance between blood coagulation and anticoagulation and causes thrombus frequently, accompanied by repeated venous thrombosis, phlebitis, and pulmonary embolism. These symptoms could lead to myocardial infarction or cerebral infarction.

When blood clots block vessels, bleeding occurs and most cases show cloudy and large purpura, like bruises, all over the skin. So far, there has been no treatment that could completely prevent or control venous thrombosis and purpura fulminant in patients with severe congenital protein C deficiency.

Ceprotin is a human protein C concentrate and the treatment was designated as an orphan drug by the Ministry of Food and Drug Safety in March 2021.

Ceprotin proved therapeutic effect against purpura fulminant and other thromboses in 18 patients with severe congenital protein C deficiency, compared to the control group treated with conventional treatment, in a clinical trial.