Drug intelligence / Profile preview

laronidase + cyclosporine A + azathioprine

Development stage
Phase 2
Lead developer
Sanofi
Modality
Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Small Molecules
Administration
Intravenous (laronidase), Oral (cyclosporine A), Oral (azathioprine)
01

Overview

This combination consists of three distinct drugs: - **Laronidase** is a recombinant form of human α-L-iduronidase used as enzyme replacement therapy for mucopolysaccharidosis type I (MPS I), including Hurler, Hurler-Scheie, and Scheie syndromes. It works by catalyzing the breakdown of glycosaminoglycans (GAGs), specifically dermatan sulfate and heparan sulfate, which accumulate due to the enzyme deficiency[1][2][4][5][6]. - **Cyclosporine A** is a calcineurin inhibitor, acting as an immunosuppressive agent. It is primarily used to prevent organ rejection in transplant recipients by inhibiting T-cell activation through blockade of interleukin-2 production. - **Azathioprine** is an immunosuppressant that interferes with purine synthesis, thereby reducing DNA, RNA, and protein synthesis in immune cells. It is used for the prevention of organ transplant rejection and for treating autoimmune diseases such as rheumatoid arthritis[3]. This particular three-drug combination does not correspond to a marketed branded product or a standard regimen but could potentially be used in clinical scenarios such as enzyme replacement therapy in a transplant recipient for MPS I requiring immunosuppression.

02

Targets

PPP3CA (Protein phosphatase 3 catalytic subunit alpha)IDUA (Alpha-L-iduronidase)

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