FDA Approves First Treatment for Congenital Thrombotic Thrombocytopenic Purpura
Takeda today announced that the U.S. Food and Drug Administration has approved Adzynma (ADAMTS13, recombinant-krhn) for the prophylactic and on-demand treatment of adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP). Adzynma is the first and only FDA approved recombinant ADAMTS13 (rADAMTS13) protein designed to address an unmet medical need in people with cTTP by replacing the deficient ADAMTS13 enzyme.
Congenital TTP is a blood disorder characterized by low platelets, low red blood cell count (caused by premature breakdown of the cells), abnormalities in kidney function, and neurological abnormalities. A similar clinical condition with overlapping features is hemolytic-uremic syndrome.
Adzynma is a purified recombinant form of the ADAMTS13 enzyme that enhances the low levels of the deficient enzyme in patients with cTTP. For prophylactic ERT, Adzynma is administered intravenously once every other week. For on-demanhttps://www.drugs.com/adzynma.htmld ERT, it is administered once daily. The most common side effects included headache, diarrhea, migraine, abdominal pain, nausea, upper respiratory tract infection, dizziness, and vomiting.
