FDA sets a Prescription Drug User Fee Act (PDUFA) target action date of April 30, 2024
If the NDA is approved, company eligible to receive a Priority Review Voucher (PRV) resulting from mavorixafor’s rare pediatric designation in WHIM syndrome
NDA supported by positive results from global, pivotal 4WHIM Phase 3 clinical trial
BOSTON, Oct. 31, 2023 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today 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 (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) 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.”
Mavorixafor is an investigational small-molecule antagonist of the CXCR4 receptor being developed as a once-daily oral therapy for WHIM syndrome and certain chronic neutropenic disorders. For the WHIM syndrome indication, mavorixafor has been granted Breakthrough Therapy Designation, Fast Track Designation, and Rare Pediatric Disease (RPD) Designation in the U.S., and Orphan Drug Status in both the U.S. and European Union. Upon FDA approval of a product with RPD designation, the sponsor can receive a Priority Review Voucher that can be used to obtain priority review for a subsequent application or sold to another drug sponsor.
The NDA is supported by the results of the global, pivotal, 4WHIM Phase 3 clinical trial of once-daily, oral mavorixafor in individuals with WHIM syndrome. The 4WHIM trial met its primary endpoint of time above threshold for absolute neutrophil count (TAT-ANC) vs. placebo (p