PANTHERx® Rare Partnering with X4 Pharmaceuticals Inc. for the Distribution of XOLREMDI™ (mavorixafor), the First FDA-Approved Therapy Indicated for Use in Patients with WHIM Syndrome
WHIM syndrome is a rare, combined primary immunodeficiency and chronic neutropenic disorder caused by reduced mobilization of white blood cells into the peripheral circulation due to CXCR4/CXCL12 pathway dysfunction.
PITTSBURGH, April 29, 2024 /PRNewswire/ -- PANTHERx® Rare, a leader in rare disease product patient access and support services in the United States, today announced it was selected by X4 Pharmaceuticals, Inc. for the distribution of XOLREMDI™ (mavorixafor), recently approved by the U.S. Food and Drug Administration (FDA) and the first FDA-approved therapy specifically indicated for use in patients 12 years of age and older with warts, hypogammaglobulinemia, infections and myelokathexis (WHIM) syndrome.
WHIM syndrome is a rare, combined primary immunodeficiency and chronic neutropenic disorder caused by reduced mobilization of white blood cells into the peripheral circulation due to CXCR4/CXCL12 pathway dysfunction. It is estimated that at least 1,000 people are currently diagnosed with WHIM syndrome in the U.S.
XOLREMDI, a once-daily oral treatment option, is a selective antagonist of the CXCR4 receptor that blocks the binding of the CXCL12 ligand to the CXCR4 receptor. By targeting the underlying cause of WHIM syndrome, treatment with XOLREMDI results in the increased mobilization of immune cells from the bone marrow.
"We are very pleased to be working with X4 Pharmaceuticals for the distribution of XOLREMDI in the U.S.," said Rob Snyder, CEO of PANTHERx Rare Pharmacy. "Up until now, there has been no treatment specifically indicated in people with WHIM syndrome. Given our focus on bringing innovative treatments to patients with rare diseases through our national distribution network, we are looking forward to partnering with X4 to have an impact on these individuals."
About PANTHERx Rare
PANTHERx Rare is a dual-accredited specialty pharmacy focused on rare and orphan disease, distributing several orphan products and providing access and support services to those needing them most. PANTHERx transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to people living with rare and devastating conditions. While each rare condition affects few people, together all rare diseases impact an estimated 25 to 30 million Americans. Currently over 7,000 rare diseases have been identified and more than 90% of rare diseases are still without an FDA-approved treatment. Changes in federal policy and advances in science have led to a surge in FDA orphan drug approvals, providing tremendous hope to the rare disease community.
PANTHERx is a dual-accredited specialty pharmacy, holding distinctions in rare disease including the Accreditation Commission for Health Care (ACHC) Distinction in Orphan Drugs and Utilization Review Accreditation Commission (URAC) Rare Disease Center of Excellence. As a pharmacy focused on patient satisfaction, PANTHERx is now a six-time winner of the prestigious MMIT Patient Choice Award, including the 2023 honor. PANTHERx is headquartered in Pittsburgh, Pennsylvania and is licensed in all 50 states and U.S. territories.
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