The U.S. FDA has greenlit Takeda’s Adzynma, a recombinant protein product for use in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), a rare and life-threatening blood clotting disorder.
cTTP, an inherited blood clotting disorder triggered by a disease-causing mutation in the ADAMTS13 gene, results in a deficiency of the ADAMTS13 enzyme, leading to the formation of blood clots in small vessels throughout the body. Without treatment, cTTP is fatal, and symptoms include severe bleeding episodes, strokes and organ damage. Adzynma, a purified recombinant form of the ADAMTS13 enzyme, offers a replacement for the deficient enzyme in patients with cTTP.
Administered intravenously, Adzynma can be used prophylactically every other week to reduce the risk of disease symptoms or on-demand daily for acute events. Its safety and effectiveness were demonstrated in a global study comparing Adzynma to plasma-based therapies in cTTP patients, where the drug proved effective in reducing the incidence of thrombotic thrombocytopenic purpura events and manifestations, as well as the need for supplemental doses.
Adzynma was reviewed with a Rare Pediatric Disease Priority Review Voucher, and it received Priority Review, Fast Track and Orphan designations from the FDA.