Drug intelligence / Profile preview

cyclosporine-A

Development stage
Discontinued
Lead developer
Novartis
Modality
Small Molecules
Administration
Ophthalmic
01

Overview

Cyclosporine-A is a small molecule cyclic polypeptide and a potent immunosuppressive agent. It functions primarily as a calcineurin inhibitor; upon entering T-cells, it binds to the cytosolic protein cyclophilin A (PPIA) to form a complex that inhibits the phosphatase activity of calcineurin. This inhibition prevents the dephosphorylation and nuclear translocation of the nuclear factor of activated T-cells (NFAT), thereby blocking the transcription of interleukin-2 (IL-2) and other cytokines essential for T-cell activation and proliferation. In a clinical research context led by Semmelweis University, a 0.5% ophthalmic solution of cyclosporine-A was investigated for the treatment of retinitis pigmentosa (RP). The study (NCT00433277) aimed to evaluate whether local immunosuppression could mitigate the progression of retinal degeneration, based on the hypothesis that autoimmune mechanisms contribute to the pathogenesis of RP.

Brand names
SandimmuneNeoralGengrafRestasisSangCyaIkervisVerkaziaAtopica
Other names
cyclosporineciclosporinCsA
02

Targets

PPIA (Peptidyl-prolyl cis-trans isomerase A)CN (Calcineurin)

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