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X4 Pharmaceuticals Receives Rare Pediatric Disease Designation from FDA for Mavorixafor for the Treatment of WHIM Syndrome
BOSTON, Dec. 10, 2020 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting
About this update from X4 Pharmaceuticals, Inc.
[{"type":"text","content":"BOSTON, Dec. 10, 2020 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, today announced that it has received Rare Pediatric Disease (RPD) Designation from the U.S. Food and Drug Administration (FDA) for its lead asset, mavorixafor, for the treatment of WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene. Mavorixafor is currently being investigated in a global pivotal Phase 3 clinical trial, 4WHIM, for the treatment of WHIM syndrome in patients who are 12 years of age and older. “WHIM is a congenital disease that affects individuals of all ages. Children, in particular, have been shown to experience serious or life-threatening bacterial infections that can require hospitalizations. There are currently no treatments available that address the underlying genetic cause of WHIM,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. “The RPD designation reinforces the clear unmet need for a disease modifying therapy in both pediatric and adult patient populations.” RPD designations are granted by the FDA for serious or life-threatening diseases in which the serious or life-threatening manifestations primarily affect individuals between birth and 18 years of age. Under the RPD program, a sponsor who receives an approval for a drug for a “rare pediatric disease” and a Fast Track designation may qualify for a voucher that can be redeemed to receive a priority review by the FDA for any subsequent marketing application for a different product. Such a voucher is transferrable and may be sold. Mavorixafor had previously been granted Fast Track Designation and Breakthrough Therapy Designation by the FDA, as well as Orphan Drug status by the FDA and the European Commission (EC), for the treatment of WHIM syndrome in adults. About Mavorixafor in WHIM SyndromeWHIM syndrome is a rare, inherited, primary immunodeficiency disease caused by gain-of-function mutations in the chemokine receptor CXCR4, resulting in a reduced mobilization and trafficking of white blood cells from the bone marrow. The company estimates there to be more than 3,500 diagnose...