Experimental WHIM Syndrome Treatment Gets FDA Fast Track Designation

According to a story from GlobeNewswire, the biopharmaceutical company X4 Pharmaceuticals, Inc. has just announced that the US Food and Drug Administration (FDA) has granted its investigational product candidate mavorixafor the Fast Track designation. This designation is for use of the drug as a treatment for WHIM syndrome in adult patients. X4 focuses on developing therapies for diseases triggered by problems with the CXCR4 pathway.

About WHIM Syndrome

WHIM syndrome, which stands for warts, hypogammaglobulinemia, immunodeficiency, and myelokathexis, is a rare, congenital syndrome that causes immunodeficiency that is characterized by chronic neutropenia, or low levels of neutrophils in the blood. This syndrome is caused by mutations that affect the CXCR4 gene. The activity of the GRK3 gene has been associated with the syndrome as well. Signs and symptoms of WHIM syndrome include the retention of neutrophils in the bone marrow, increased vulnerability to bacterial and viral infections (especially HPV), deficiencies in IgG antibodies and lymphocytes, and warts on the hands and feet. Treatment is mostly symptomatic and includes therapies to reduce bacterial infections and improve counts of neutrophils in the blood. There is currently no known cure. Research studies have suggested that CXCR4 antagonists could be an effective approach. To learn more about WHIM syndrome, click here.

About Fast Track Designation

Fast Track designation is reserved for therapies that have demonstrated the potential to fulfill an unmet medical need in severe, life-threatening diseases. The designation is intended to speed up the development process and X4 will be permitted to have more meetings and collaboration with the FDA in order to facilitate its approval.

About Mavorixafor

Mavorixafor is a first in its class antagonist of the CXCR4 chemokine receptor. The drug is currently being evaluated in a phase 3 clinical trial that is expected to include a total of 28 adult patients. The therapy has demonstrated potential therapeutic benefit in previous evaluations. Mavorixafor has also earned Breakthrough Therapy designation in the US as well as Orphan Drug designation in the US and EU. 

Patients living with WHIM syndrome are currently forced to manage their disease without access to a treatment that addresses the root cause. Mavorixafor has the potential to be a real game changer in treating this disease if it continues to be effective in trials.

Follow us