(Washington, D.C.) – The U.S. Food and Drug Administration approved Adzynma, the first recombinant (genetically engineered) protein product indicated for prophylactic (preventive) or on demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), a rare and life-threatening blood clotting disorder.
It is estimated that cTTP affects fewer than one thousand people in the United States. Symptoms typically develop in infancy or early childhood, but in some cases may develop in adulthood and may first manifest during pregnancy.
