X4 Pharmaceuticals Receives Orphan Drug Designation from European Commission for Mavorixafor for Treatment of WHIM Syndrome
X4 Pharmaceuticals, Inc. (XFOR)
Last x4 pharmaceuticals, inc. earnings: 3/12 07:30 am
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Source: Business Wire
CAMBRIDGE, Mass.--(BUSINESS WIRE)--X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a clinical-stage biopharmaceutical company focused on the development of novel therapeutics for the treatment of rare diseases, today announced that the European Commission (EC), based on a favorable recommendation from the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP), has granted orphan drug designation (ODD) to mavorixafor (X4P-001) for the treatment of WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene. In October 2018, mavorixafor was granted ODD for the treatment of WHIM syndrome by the U.S. Food and Drug Administration. The exact prevalence of WHIM syndrome is unknown, however, in the United States alone there are between 15,000 and 100,000 patients classified as having a primary immunodeficiency disease of unknown origin – of which W
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