X4 Pharma’s mavorixafor gets EC orphan drug designation for WHIM syndrome

The European Commission (EC) granted X4 Pharmaceuticals’ (NASDAQ:XFOR) mavorixafor orphan drug designation for WHIM syndrome.

WHIM stands for warts; hypogammaglobulinemia, or low antibody levels; infections; and myelokathexis, or low white blood cell levels. The syndrome is an inherited immunodeficiency disease caused by mutations in the CXCR4 receptor gene. Mavorixafor is an orally administered CXCR4 receptor antagonist.

“We look forward to advancing mavorixafor through our recently commenced Phase 3 pivotal trial and bringing this drug to patients,” Paula Ragan, X4’s president and CEO, said in a statement.

Mavorixafor received FDA orphan drug designation in October 2018.


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