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.
For more information, please contact
marketing@pantherxrare.com
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SOURCE PANTHERx Rare Pharmacy