X4 Pharmaceuticals’ lead product candidate, Mavorixafor (X4P-001) has reached a significant milestone with the submission of a New Drug Application (NDA) to the U.S. FDA for the treatment of WHIM Syndrome on September 5. This marks an important step forward in the regulatory approval process for Mavorixafor and highlights its potential as a promising therapy for patients with WHIM Syndrome.
WHIM syndrome was named after four symptoms: Warts, Hypogammaglobulinemia, Infections, Myelokathexis. WHIM syndrome is a rare and difficult-to-diagnose primary immunodeficiency in which the body’s immune system does not function properly and has trouble fighting infections, especially respiratory, ear, and skin infections.
Mavorixafor is an investigational small-molecule CXCR4 antagonist. It allosterically blocks the overstimulation of the CXCL12/CXCR4 pathway, having the potential to improve immune cell trafficking and immunosurveillance. Mavorixafor was designated orphan drug status by both the U.S. and the EU, and previously granted Fast Track Designation and Breakthrough Therapy Designation by the FDA.