Paula Ragan, President and Chief Executive Officer of X4 Pharmaceuticals, discusses the pathway of approval under the European Medicines Agency (EMA) for mavorixafor for patients with WHIM syndrome.
WHIM syndrome is an acronym for a rare immunodeficiency disorder with each letter representing a predominant feature of the condition: warts, hypogammaglobulinemia, infections, and myelokathexis. Symptoms usually begin in early childhood with recurrent bacterial infections such as respiratory infections and cellulitis. It is caused by genetic changes in the CXCR4 gene.
Mavorixafor is an oral, once-daily selective CXCR4 receptor antagonist approved in the U.S. for the treatment of patients 12 years and older with WHIM syndrome. The 4WHIM phase 3 clinical trial met its primary endpoint, a key secondary endpoint, was generally well-tolerated, and exhibited no serious treatment-related adverse events. It was also observed to reduce the rate, severity, and duration of infections in patients with WHIM syndrome. Additionally, the treatment is also in development for chronic neutropenia, including a phase 3 clinical trial.
Recently, a submission of a Marketing Authorization Application (MAA) to the EMA has been approved and is currently being evaluated under the EMA’s Committee for Medicinal Products for Human Use (CHMP). X4 has entered an exclusive licensing and supply agreement to commercialize mavorixafor in Europe, Australia, and New Zealand following EMA approval.
For more information, click here.
To learn more about WHIM and other rare genetic conditions, visit https://checkrare.com/diseases/congenital-and-genetic-conditions/