US FDA approves X4 Pharmaceuticals' therapy for rare immunodeficiency disease

X4 Pharmaceuticals: X4 Pharmaceuticals has received approval from the US Food and Drug Administration (FDA) for its therapy to treat a rare genetic immunodeficiency disease known as WHIM syndrome in patients aged 12 years and older. The announcement led to a significant surge in X4 Pharmaceuticals' shares, which rose by 16 per cent before the market opened on Monday.

The therapy, named mavorixafor, will be marketed under the brand name Xolremdi. It marks a significant milestone as the first treatment to receive US approval specifically for WHIM syndrome.

WHIM syndrome is a rare condition characterised by various immune system dysfunctions, including warts, hypogammaglobulinemia (low antibody levels), infections, and myelokathexis (low white blood cell count). These symptoms collectively compromise the body's ability to defend against infections and illnesses.

The FDA's decision to approve mavorixafor was based on positive results from a late-stage clinical trial. The study demonstrated a remarkable 60 per cent reduction in infection rates compared to a placebo among patients with WHIM syndrome.

According to government data, WHIM syndrome affects fewer than 1,000 individuals in the United States. This underscores the rarity of the condition and the urgent need for effective treatment options.

X4 Pharmaceuticals' achievement in gaining FDA approval for mavorixafor represents a significant advancement in the field of rare disease therapeutics. The therapy holds promise in improving the quality of life for individuals living with WHIM syndrome by addressing its underlying immune system abnormalities.

(With Reuters inputs.)