Posted by Michael Wonder on 12 Nov 2019
X4 Pharmaceuticals receives breakthrough therapy designation from the FDA for mavorixafor for the treatment of WHIM syndrome
12 November 2019 - X4 Pharmaceuticals today announced that the U.S. FDA has granted breakthrough therapy designation for mavorixafor (X4P-001) for the treatment of adult patients with WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene.
Mavorixafor is a potential first-in-class, once-daily, oral, small molecule antagonist of chemokine receptor CXCR4, and is currently being investigated in a pivotal Phase 3 global clinical trial, 4WHIM, for the treatment of WHIM syndrome.
The breakthrough therapy designation granted to mavorixafor is based on data from X4’s Phase 2 open-label, multi-center trial of mavorixafor in adult patients with WHIM syndrome. In this trial, proof of concept was established based on clinically meaningful increases in absolute neutrophil counts, absolute lymphocyte counts, evidence indicating reductions in infection rates and wart burden, and a safety profile showing that mavorixafor is well-tolerated.
