Further controlled assessment of the safety and efficacy of plerixafor in patients with WHIM syndrome is a challenge because the disease is extremely rare. 4 Nevertheless, our phase 3 trial of G ...

New data related to clinical secondary endpoints, among other assessments, are to be presented ... for mavorixafor for the treatment of WHIM syndrome, which is expected early in the second ...

The EMA previously granted orphan designation to mavorixafor in WHIM syndrome. In April 2024, mavorixafor received U.S. Food and Drug Administration approval as XOLREMDI ®, an oral, once-daily ...