On April 29, 2024, X4 Pharmaceuticals, Inc. (XFOR) announced that the U.S. Food and Drug Administration (FDA) has approved XOLREMDI (mavorixafor) for use in patients 12 years of age and older with WHIM syndrome. This is the first therapy specifically indicated for WHIM syndrome. X4 also received a Rare Pediatric Disease Priority Review Voucher (PRV), which we anticipate the company monetizing in the near future (multiple PRVs have recently sold for approximately $100 million each). The company has established a price of approximately $496,000/yr for those > 50 kg in weight (estimated to be ~90% of patients) and approximately $372,000/yr for those = 50 kg in weight. X4 has a comprehensive plan in place for commercialization, which includes a dedicated sales force, patient/physician education, and gaining broad payer access.
30 Apr 2024
XFOR: XOLREMDI Approved by FDA
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XFOR: XOLREMDI Approved by FDA
- Published:
30 Apr 2024 -
Author:
David Bautz -
Pages:
5
On April 29, 2024, X4 Pharmaceuticals, Inc. (XFOR) announced that the U.S. Food and Drug Administration (FDA) has approved XOLREMDI (mavorixafor) for use in patients 12 years of age and older with WHIM syndrome. This is the first therapy specifically indicated for WHIM syndrome. X4 also received a Rare Pediatric Disease Priority Review Voucher (PRV), which we anticipate the company monetizing in the near future (multiple PRVs have recently sold for approximately $100 million each). The company has established a price of approximately $496,000/yr for those > 50 kg in weight (estimated to be ~90% of patients) and approximately $372,000/yr for those = 50 kg in weight. X4 has a comprehensive plan in place for commercialization, which includes a dedicated sales force, patient/physician education, and gaining broad payer access.