X4 Pharmaceuticals Announces Submission of New Drug Application (NDA) to U.S. FDA for Mavorixafor in WHIM Syndrome
“The submission of our first NDA is a significant milestone in X4's journey to transform the care of those living with rare immunodeficiencies,” said
The FDA takes 60 days to determine whether an NDA is sufficiently complete prior to accepting it for filing. X4 has requested priority review for the application which, if granted, would provide a target FDA review period of six months from the application acceptance for filing date.
The NDA submission 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 and key secondary endpoint and was generally well tolerated in the trial, with no treatment-related serious adverse events reported and no discontinuations for safety events. The 4WHIM data also revealed that mavorixafor treatment resulted in reductions in the rate, severity, and duration of infections in trial participants versus placebo. These and additional 4WHIM Phase 3 data were published in oral presentations at the annual meetings of both the
About Mavorixafor and WHIM Syndrome
WHIM syndrome is a rare, inherited, combined immunodeficiency disease caused by reduced mobilization and trafficking of white blood cells from the bone marrow due to over-signaling of the CXCR4/CXCL12 pathway. WHIM syndrome is named for its four common clinical findings: Warts, Hypogammaglobulinemia, Infections, and Myelokathexis, although not all patients experience all symptoms, and not all symptoms are required for a diagnosis. People with WHIM syndrome characteristically have very low blood levels of neutrophils (neutropenia) and lymphocytes (lymphopenia), and as a result, experience frequent, recurrent infections with a high risk of lung disease, refractory warts from underlying human papillomavirus (HPV) infection, limited antibody production due to low levels of immunoglobulin, and an increased risk of developing certain types of cancer.
Mavorixafor is an investigational small-molecule antagonist of CXCR4 being developed as a once-daily oral therapy for WHIM syndrome. For the WHIM syndrome indication, mavorixafor has been granted Breakthrough Therapy Designation, Fast Track Designation, and Rare Pediatric Designation in the
About the 4WHIM Phase 3 Clinical Trial
The 4WHIM Phase 3 clinical trial was a global, randomized, double-blind, placebo-controlled, multicenter study designed to evaluate the efficacy and safety of oral, once-daily mavorixafor in people with genetically confirmed WHIM syndrome. The trial enrolled 31 participants aged 12 and older who received either mavorixafor (n=14) or placebo (n=17) orally once daily for 52 weeks. An open-label extension phase of the clinical trial is ongoing (NCT03995108).
This press release contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target,” or other similar terms or expressions that concern X4's expectations, strategy, plans, or intentions. Forward-looking statements include, without limitation, statements regarding the FDA’s review of the NDA for mavorixafor in WHIM syndrome, if accepted, and commercial launch of mavorixafor, if approved. Any forward-looking statements in this press release are based on management's current expectations and beliefs. Actual events or results may differ materially from those expressed or implied by any forward-looking statements contained herein, including, without limitation, the risk that the FDA does not accept the NDA submission for mavorixafor for the treatment of WHIM syndrome; the risk that such NDA is not approved by the FDA or such approval is delayed; the risk that commercial launch of mavorixafor in WHIM syndrome, if approved, is delayed; the risk that the potential market performance for mavorixafor, if approved, may differ materially from projections; and other risks and uncertainties, including those described in the section entitled “Risk Factors” in X4’s Quarterly Report on Form 10-Q filed with the
Source: X4 Pharmaceuticals