X4 announces FDA acceptance with priority review of U.S. NDA for mavorixafor

X4 Pharmaceuticals announced that the United States Food and Drug Administration, FDA, has accepted for filing the company’s New Drug Application, NDA, for once-daily, oral mavorixafor to treat individuals aged 12 and older with WHIM syndrome, a rare, primary immunodeficiency. The FDA granted Priority Review of the mavorixafor NDA, establishing a goal of six months review from the date of acceptance and assigning a Prescription Drug User Fee Act, PDUFA, target action date of April 30, 2024. “The FDA’s acceptance of our mavorixafor NDA with priority review represents yet another significant step forward towards a potential treatment for those with WHIM syndrome, a rare disease for which there are currently no approved therapies,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. “We look forward to working closely with the FDA throughout the NDA review process with the goal of bringing mavorixafor to people with WHIM syndrome as quickly as possible.”