The U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to mavorixafor for WHIM syndrome and evaluated the New Drug Application under Priority Review. It is approved for patients aged 12 and older with WHIM syndrome (warts, hypogammaglobulinemia, infections, and myelokathexis) to increase circulating mature neutrophils and lymphocytes. Individuals with WHIM syndrome typically have neutropenia and lymphopenia in their blood and suffer from serious and/or frequent infections.
Mavorixafor, a selective CXC chemokine receptor 4 (CXCR4) antagonist, is the first therapy specifically indicated for WHIM syndrome. Clinical Trial The approval of mavorixafor is based on the pivotal 4WHIM Phase 3 clinical trial, a global, randomized, double-blind, placebo-controlled, 52-week multicenter study. It involved 31 patients aged 12 and older with WHIM syndrome. The study…
