Patients in the US with the ultra-rare disease WHIM syndrome now have their first approved treatment, after X4 Pharma’s Xolremdi was given a green light by the FDA.
Oral CXCR4 antagonist Xolremdi (mavorixafor) can be used in patients aged 12 and over with WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome, a congenital immunodeficiency characterised by low neutrophil counts that affects fewer than 1,000 people in the US.
