Enzyme replacement therapy

• The first and only enzyme replacement therapy for the treatment of a very rare and complex blood disorder has been approved by the US Food and Drug Administration.
• Adzynma, the first recombinant (genetically engineered) protein product indicated for on-demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP) has been approved for Takeda Pharmaceuticals U.S.A. Inc.

ABOUT ERT

• Enzyme replacement therapy is typically used to replace a missing or deficient enzyme in a person with an inherited enzyme deficiency syndrome.
• The replacement enzyme derived from human, animal or plant cells is administered directly into the bloodstream of patient through fluids.

ABOUT CTTP

• cTTP is a rare and life-threatening blood clotting disorder.
• It is caused by a disease-causing mutation in the ADAMTS13 gene, which is responsible for making an enzyme, also named ADAMTS13, that regulates blood clotting.
• A deficiency in this enzyme causes blood clots to form in the small blood vessels throughout the body.
• Symptoms of this rare disorder typically develop in infancy or early childhood, but in some cases may develop in adulthood and may first manifest during pregnancy.
• Individuals with cTTP may experience severe bleeding episodes, strokes and damage to vital organs.
• If left untreated, the disease can be fatal.