Dosing & Uses
Hematopoietic Stem Cell Transplant-associated Thrombotic Microangiopathy
Pending FDA approval for hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA)
Pharmacology
Mechanism of Action
Human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of the complement system
Lectin pathway is a principal pathway of complement and is activated primarily by tissue damage and microbial infection; notably, MASP-2 inhibition appears not to interfere with the complement pathway, an essential component of the acquired immune response to infection
The drug is designed to prevent complement-mediated inflammation and endothelial damage while leaving intact the respective functions of the other pathways of innate immunity