X4 Pharmaceuticals announced today that the U.S. Food and Drug Administration has approved XOLREMDI™ capsules for patients aged 12 and above diagnosed with WHIM syndrome (warts, hypogammaglobulinemia, infections, and myelokathexis). XOLREMDI, a selective CXC chemokine receptor 4 (CXCR4) antagonist, marks a breakthrough as the first therapy specifically tailored for individuals with WHIM syndrome, a rare immunodeficiency disorder stemming from CXCR4 pathway dysfunction. Patients with WHIM syndrome face low levels of neutrophils and lymphocytes in their blood, making them susceptible to severe and recurrent infections.

X4 Pharmaceuticals, expressed gratitude to the WHIM syndrome community, clinical investigators, U.S. regulators, and dedicated staff for realizing this pivotal therapy. Jorey Berry, CEO of the Immune Deficiency Foundation (IDF), emphasized the importance of innovative treatments for primary immunodeficiencies and hailed the approval of XOLREMDI as a significant advancement.

Duke University School of Medicine and a principal investigator in the 4WHIM trial, hailed XOLREMDI as a targeted treatment addressing the root cause of WHIM syndrome. The FDA's approval was based on the positive outcomes of the pivotal 4WHIM Phase 3 clinical trial, showcasing XOLREMDI's efficacy in boosting neutrophil and lymphocyte counts while reducing infections.

With XOLREMDI's approval, X4 has received a Rare Pediatric Disease Priority Review Voucher, offering priority review for future applications or potential sale to another drug sponsor.

X4 also introduced X4Connect™ to support WHIM syndrome patients in accessing XOLREMDI, offering resources, insurance navigation, and copay assistance. XOLREMDI will be available through X4's specialty pharmacy partner, PANTHERx® Rare.

WHIM syndrome, characterized by CXCR4 receptor dysfunction, leads to impaired white blood cell mobilization and low blood cell counts, predisposing patients to infections. XOLREMDI, a selective CXCR4 receptor antagonist, aids in increasing circulating neutrophils and lymphocytes.

This approval marks a significant stride in addressing the unmet needs of WHIM syndrome patients and underscores X4 Pharmaceuticals' commitment to advancing rare disease treatments.

 

Source: globenewswire.com

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