X4 cues up FDA verdict in April for WHIM syndrome drug

The FDA has started a priority review of X4 Pharma’s mavorixafor, angling to become the first therapy for the rare disease WHIM syndrome, with a decision due by 30th April next year.

WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome is a rare congenital immunodeficiency that affects fewer than 1,000 people in the US and is caused by a mutation in the gene for CXCR4, a chemokine receptor involved in immune functioning.