mavorixafor

Mavorixafor is an orally bioavailable small molecule that acts as a selective antagonist of the CXC chemokine receptor 4 (CXCR4). It is used to treat WHIM syndrome (warts, hypogammaglobulinemia, infections, and myelokathexis), a rare genetic immunodeficiency disorder caused by gain-of-function mutations in the CXCR4 gene. These mutations lead to increased responsiveness to its ligand CXCL12 and retention of leukocytes in the bone marrow. By blocking the binding of CXCL12 to CXCR4, mavorixafor inhibits this pathway and increases mobilization of neutrophils and lymphocytes from bone marrow into peripheral blood, thereby improving immune function. Mavorixafor was developed by X4 Pharmaceuticals and is marketed under the brand name Xolremdi for patients aged 12 years or older with WHIM syndrome[1][2][3][5][7][8].

Brand names

Xolremdi

Other names

mavorixafor