Drug intelligence / Profile preview

cyclophosphamide + tacrolimus + mycophenolate mofetil

Development stage
Unknown
Lead developer
Takeda
Modality
Small Molecules
Administration
Intravenous, Oral
01

Overview

This pharmacological combination consists of three **immunosuppressive agents**—**cyclophosphamide**, **tacrolimus**, and **mycophenolate mofetil**—administered together primarily as prophylaxis against graft-versus-host disease (GVHD) in allogeneic hematopoietic stem cell transplantation (HSCT), and in severe autoimmune disorders such as systemic lupus erythematosus (SLE)[3][5][1]. - **Cyclophosphamide** is an alkylating agent that crosslinks DNA, resulting in cytotoxicity to rapidly dividing lymphocytes and immunosuppressive effects. - **Tacrolimus** is a calcineurin inhibitor that blocks T-cell activation by inhibiting interleukin-2 gene transcription. - **Mycophenolate mofetil** inhibits inosine monophosphate dehydrogenase in lymphocytes, blocking purine synthesis essential for cell proliferation. The combination is designed to synergistically suppress immune function, reduce the risk of GVHD post-transplant, and enhance renal response in autoimmune conditions[1][3][5]. There is robust evidence, including phase III randomized clinical trials, showing this regimen is associated with lower rates of GVHD and improved transplant outcomes compared to standard combinations, such as tacrolimus-methotrexate[3]. It is also used off-label in refractory severe autoimmune disease populations where intensive immunosuppression is required[1].

Other names
PTCy/Tac/MMFPost-transplant cyclophosphamide, tacrolimus, and mycophenolate mofetil
02

Targets

IMPDH (Inosine-5'-monophosphate dehydrogenase 1)FKBP1ADNACN (Calcineurin)

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