GC Biopharma announced that GC1126A, a drug candidate for thrombotic thrombocytopenic purpura (TTP), has received orphan drug designation from the U.S. Food and Drug Administration (FDA).
GC1126A, a novel ADAMTS13 mutein, is specifically engineered to circumvent autoantibodies while boasting an extended half-life. GC1126A has maintained higher activity levels when compared to both existing treatments and the natural wild-type ADAMTS13 enzyme.
TTP is a rare and life-threatening blood disorder. It is characterized by the formation of small blood clots throughout the body, which can obstruct blood flow to critical organs like the brain and heart.
The condition is associated with a deficiency of the proteolytic enzyme ADAMTS13 in the patient's body (congenital TTP) or the sudden production of antibodies that inhibit the enzyme's activity (immune-mediated TTP). If left untreated, TTP can have a high mortality rate, reaching up to 90 percent.
The orphan drug designation for GC1126A suggests that it shows promise as a potential treatment for TTP, a rare and life-threatening condition
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