| Full Name | Adzynma (ADAMTS13, recombinant-krhn) |
| Drug | Adzynma |
| Manufacturer | Takeda Pharmaceuticals U.S.A., Inc. |
| Route of Administration | Intravenous |
| Site of Care | Home or Healthcare Facility |
| Approved Indication | Prophylactic or on demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP) |
| Disease | Congenital thrombotic thrombocytopenic purpura (c-TTP) |
| Therapeutic Area | Hematology |
| Enrollment Form Link | • Orsini Prescription Form • Adzynma Patient Support Enrollment Form (optional) |
| Phone Number | 800-720-0789 |
| Fax Number | 877-251-0709 |
| Product Website | adzynma.com |
About Congenital Thrombotic Thrombocytopenic Purpura (c-TTP)
Congenital thrombotic thrombocytopenic purpura (c-TTP) is a rare and inherited blood disorder characterized by deficient activity of the enzyme ADAMTS13. This leads to the formation of blood clots in small blood vessels throughout the body, causing thrombocytopenia (low platelet count) and other complications. The lack of properly functioning ADAMTS13 impairs the breakdown of a specific blood clotting factor, resulting in the formation of microclots. c-TTP’s symptoms include purpura, neurological abnormalities, and organ damage. This lifelong condition poses challenges in maintaining blood clotting balance.



















