Drug intelligence / Profile preview

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

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

Coagulation Factor IXaCD20 (B-lymphocyte antigen CD20)CN (Calcineurin)Cyp (Cyclophilin family)EPCR (Endothelial protein C receptor)F9 (Coagulation Factor IX)F10 (Factor Xa)IMPDH (Inosine-5'-monophosphate dehydrogenase 1)F2 (Thrombin)F3 (Tissue factor)AVPR2 (Arginine vasopressin V2 receptor)BB-031 (Von Willebrand factor)F8 (Coagulation Factor VIIIa)

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