FDA Approves X4 Pharmaceuticals' Mavorixafor As First Therapy For Rare Immunodeficiency Disorder
Portfolio Pulse from Vandana Singh
The FDA has approved X4 Pharmaceuticals Inc's (NASDAQ:XFOR) Xolremdi (mavorixafor) for patients 12 and older with WHIM syndrome, marking it as the first therapy for this rare immunodeficiency disorder. The approval is based on Phase 3 trial results showing significant improvements in neutrophil and lymphocyte counts and a reduction in infections. X4 Pharmaceuticals has also received a Rare Pediatric Disease Priority Review Voucher and set the annual cost of Xolremdi at $496,400 for patients over 50 kg and $372,300 for those 50 kg or under. Analysts predict the therapy could generate $54.6 million in sales by 2024, potentially growing to $450.9 million by 2027.
April 29, 2024 | 4:10 pm
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X4 Pharmaceuticals Inc receives FDA approval for Xolremdi, the first therapy for WHIM syndrome. The approval could significantly impact sales, with projections of $54.6 million by 2024 and potential growth to $450.9 million by 2027.
The FDA approval of Xolremdi for WHIM syndrome represents a significant milestone for X4 Pharmaceuticals, marking it as the first therapy for this rare disorder. The approval is based on positive Phase 3 trial results, demonstrating the drug's efficacy in improving patient outcomes. The company's receipt of a Rare Pediatric Disease Priority Review Voucher and the setting of Xolremdi's annual cost suggest strong revenue potential. Analyst projections of sales reaching $54.6 million by 2024, with a potential increase to $450.9 million by 2027, indicate a positive outlook for XFOR's financial performance and stock price in the short term.
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