On Nov. 9, 2023, the U.S. Food and Drug Administration approved ‘ Takeda PharmaceuticalsVAdzynma, 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.

The very rare, inherited blood clotting disorder called cTTP 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.