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X4 Gets First Approval In Ultra-Rare Disorder, Needs Time To Build A Market

 
• By Joseph Haas

Xolremdi was OKed as the first drug for WHIM syndrome and X4 set pricing at $372,000-$496,000 a year, but the drug’s bigger opportunity may be in chronic neutropenia.

FDA Approved
X4 gets its first drug approval, in ultra-rare WHIM syndrome

With its first drug approval and the first approved therapy for the ultra-rare immunodeficiency disorder WHIM syndrome, X4 Pharmaceuticals, Inc. told a 29 April investor call that while Xolremdi (mavorixafor) will be available immediately, it sees 2024 as a year for laying the groundwork for clinician understanding and payer acceptance of the drug.

Key Takeaways

  • About two years after a 20% staff cut, X4 gets its first FDA approval for Xolremdi in the rare immunodeficiency disorder WHIM syndrome.

  • The...

Xolremdi is a once-daily capsule indicated in the US as a 400mg dose in patients weighing more than 50kg and dosed at 300mg in patients at or below 50kg. It...

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