regulatory
confidence high
sentiment positive
materiality 0.90
X4 Pharma receives FDA approval for XOLREMDI (mavorixafor), first drug for WHIM syndrome
X4 Pharmaceuticals, Inc
- XOLREMDI approved for patients 12+ with WHIM syndrome to increase circulating neutrophils and lymphocytes.
- Phase 3 4WHIM trial met primary endpoint (TAT-ANC) vs. placebo (p<0.0001).
- XOLREMDI showed 60% reduction in annualized infection rate vs. placebo.
- X4 granted Rare Pediatric Disease Priority Review Voucher concurrent with approval.
- U.S. commercial launch via specialty pharmacy PANTHERx Rare; X4Connect patient support launched.