FDA grants RPD designation for mavorixafor as WHIM syndrome therapy

11 December 2020

Shares of US biotoech firm X4 Pharmaceuticals (Nasdaq: XFOR) closed down 2.56% at $6.10 yesterday, despite the company gaining a coveted regulatory designation for its lead drug candidate mavorixafor.

X4 pharma announced that the US Food and Drug Administration has granted Rare Pediatric Disease (RPD) designation for mavorixafor, for the treatment of WHIM (Warts, Hypogammaglobulinemia, Infections and Myelokathexis) syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene.

Mavorixafor is currently being investigated in a global pivotal Phase III clinical trial, 4WHIM, for the treatment of WHIM syndrome in patients who are 12 years of age and older.

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