10 December 2020 - X4 Pharmaceuticals today announced that it has received rare paediatric disease designation from the U.S. FDA for its lead asset, mavorixafor, 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.
Mavorixafor is currently being investigated in a global pivotal Phase 3 clinical trial, 4WHIM, for the treatment of WHIM syndrome in patients who are 12 years of age and older.