FDA Grants Multiple Approvals to Takeda for Rare Blood Disorders and Cancer Treatments
The FDA has approved Adzynma, the first recombinant protein product for the treatment of congenital thrombotic thrombocytopenic purpura (cTTP), a rare and life-threatening blood clotting disorder. cTTP is caused by a mutation in the ADAMTS13 gene, resulting in a deficiency of the ADAMTS13 enzyme responsible for regulating blood clotting. Adzynma is a purified recombinant form of the enzyme that can be administered prophylactically or on-demand to reduce the risk of clotting/bleeding and treat acute events. The safety and effectiveness of Adzynma were demonstrated in a global study, and the most common side effects include headache, diarrhea, and abdominal pain. The approval of Adzynma provides a much-needed treatment option for patients with cTTP.