pharmaphorum April 29, 2024
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.
It is caused by a mutation in the gene for CXCR4, a chemokine receptor involved in immune functioning, and as its name suggests leads to warts, recurrent infections and bone marrow problems, as well as some forms of cancer.
In the phase 3 4WHIM study, which enrolled 31 adolescents and adults with the...
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