XFOR - FDA Approves X4 Pharmaceuticals' Mavorixafor As First Therapy For Rare Immunodeficiency Disorder | Benzinga
The FDA approved on Monday X4 Pharmaceuticals Inc’s (NASDAQ:XFOR) Xolremdi (mavorixafor) capsules for use in patients 12 years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis) to increase the number of circulating mature neutrophils and lymphocytes.
Xolremdi, a selective CXC chemokine receptor 4 (CXCR4) antagonist, is the first therapy specifically indicated in patients with WHIM syndrome, a rare, combined primary immunodeficiency and chronic neutropenic disorder caused by CXCR4 pathway dysfunction.
People with WHIM syndrome characteristically have low blood levels of neutrophils (neutropenia) and lymphocytes (lymphopenia) and experience serious and/or frequent infections.
The FDA approval of Xolremdi was based on 4WHIM Phase 3 trial results.
The efficacy of Xolremdi was determined by improvement in absolute neutrophil counts (ANC), ...