{"entity":{"id":"mavorixafor","kind":"drug","name":"Mavorixafor","aka":["X4P-001","AMD-070"],"tldr":"Mavorixafor is a once-daily tablet that blocks CXCR4, a receptor that traps white cells in the bone marrow. It is approved for the rare immunodeficiency WHIM syndrome and is being tested in Waldenström macroglobulinaemia, where CXCR4 mutations blunt the response to ibrutinib.","summary":"Mavorixafor is X4 Pharmaceuticals' oral CXCR4 antagonist. The FDA approved it in April 2024 for WHIM syndrome, a rare primary immunodeficiency caused by gain-of-function CXCR4 mutations, where it raises neutrophil and lymphocyte counts and reduces infections. The same mutations occur somatically in 30 to 40 percent of Waldenström macroglobulinaemia, where they lower and slow responses to BTK inhibitors.\n\nA phase 1b trial combined mavorixafor with ibrutinib in CXCR4-mutant Waldenström macroglobulinaemia and reported deeper responses than expected from ibrutinib alone in this subgroup. The Waldenström page names the trials as one answer to the open problem of CXCR4-mutant disease.","status":"approved","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Mavorixafor","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Mavorixafor"}],"tags":["subtype-drugs-wave"],"related":["ibrutinib"],"cancers":["waldenstrom"],"sections":[],"technologies":[],"targets":["cxcr4"],"drugs":[],"companies":["x4-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Xolremdi","code":"X4P-001","modality":"Oral CXCR4 antagonist","mechanism":"Blocks the chemokine receptor CXCR4, releasing neutrophils and lymphocytes from the marrow; in Waldenström macroglobulinaemia, activating CXCR4 mutations in about a third of patients cause resistance to BTK inhibitors, which the antagonist is meant to overcome.","approvals":[{"region":"US","year":2024,"indication":"WHIM syndrome (warts, hypogammaglobulinaemia, infections and myelokathexis) in patients aged 12 and over"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},"route":"/drugs/mavorixafor/","neighbours":{"drug":[{"id":"ibrutinib","kind":"drug","name":"Ibrutinib","route":"/drugs/ibrutinib/"}],"cancer":[{"id":"waldenstrom","kind":"cancer","name":"Waldenström macroglobulinaemia","route":"/cancers/waldenstrom/"}],"target":[{"id":"cxcr4","kind":"target","name":"CXCR4","route":"/targets/cxcr4/"}],"company":[{"id":"x4-pharmaceuticals","kind":"company","name":"X4 Pharmaceuticals","route":"/companies/x4-pharmaceuticals/"}]}}