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rFVIIIa + activated prothrombin complex concentrate + factor VIII + desmopressin + azathioprine + rituximab + cyclosporin

Development stage
Unknown
Lead developer
Novo Nordisk
Modality
Monoclonal Antibodies → Antibody-Based Therapeutics, Recombinant Proteins and Enzymes, Small Molecules
Administration
Intravenous, Oral, Subcutaneous
01

Overview

This is a **combination therapy** comprising seven pharmacological agents: recombinant activated factor VIII (rFVIIIa), activated prothrombin complex concentrate (aPCC), factor VIII, desmopressin, azathioprine, rituximab, and cyclosporin. The combination's primary clinical utility is in the management of **refractory bleeding in hemophilia with inhibitors** (using rFVIIIa, aPCC, factor VIII, and desmopressin) and for **immunosuppression** in autoimmune or transplant-related conditions (using azathioprine, rituximab, and cyclosporin)[1][4][7][9]. - **rFVIIIa, aPCC, and factor VIII** are procoagulant agents employed to promote hemostasis. **Desmopressin** increases endogenous factor VIII and von Willebrand factor levels. **Azathioprine, rituximab, and cyclosporin** are immunosuppressive drugs with different mechanisms—azathioprine is an antimetabolite, rituximab is a CD20-directed monoclonal antibody, and cyclosporin is a calcineurin inhibitor. - Sequential and combination use of rFVIIIa and aPCC is employed in patients with hemophilia and inhibitors when monotherapy is insufficient, but this approach is reserved for settings with close monitoring due to thrombosis risk[1][4][7][10]. - The combination of immunosuppressants (azathioprine, rituximab, cyclosporin) is used in various settings for immune modulation[3][6][9].

02

Targets

CD20 (B-lymphocyte antigen CD20)F10 (Factor Xa)BB-031 (Von Willebrand factor)EPCR (Endothelial protein C receptor)AVPR2 (Arginine vasopressin V2 receptor)F2 (Thrombin)F9 (Coagulation Factor IX)IMPDH (Inosine-5'-monophosphate dehydrogenase 1)Cyp (Cyclophilin family)F3 (Tissue factor)CN (Calcineurin)Coagulation Factor IXa

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