PITTSBURGH, Pa., July 25,
2024 /PRNewswire/ -- PANTHERx® Rare, a leader in rare
disease product patient access and support services in the
United States, is pleased to
announce it is partnering with ITF Therapeutics LLC for the U.S.
distribution of DUVYZAT™ (givinostat), the first
nonsteroidal drug approved to treat patients with all genetic
variants of Duchenne muscular dystrophy (DMD) aged six years and
older.
DUVYZAT, a twice daily oral suspension, is a histone deacetylase
(HDAC) inhibitor that reduces inflammation and loss of muscle
observed in DMD.
DMD is a rare and serious muscle disorder affecting
approximately 1 in 5,000 male births worldwide and is caused by a
variant in the DMD gene located on the X chromosome. This gene is
responsible for regulating the production of dystrophin, which is a
protein located in skeletal and cardiac muscle cells. Disease onset
in DMD is typically observed between three and five years of age
and progression is characterized by muscle wasting and weakness in
the voluntary muscles of the body. The disease typically affects
muscles closest to the trunk (e.g., upper legs, upper arms,
shoulders, and pelvic muscles) first and then progresses outward
toward the extremities. By late teenage years, many patients
develop additional potentially life-threatening complications
including cardiomyopathy and respiratory failure.
"We are looking forward to serving the needs of people living
with Duchenne muscular dystrophy and their families and caregivers
by streamlining the process associated with getting rare
medications from the people who create them to the people who need
them most. We appreciate ITF Therapeutics' commitment to making a
difference in the lives of patients with this disease," said
Rob Snyder, CEO of PANTHERx® Rare
Pharmacy.
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 US territories.
About DUVYZAT™ (givinostat)
DUVYZAT is a U.S. FDA
approved therapy indicated for the treatment of patients six years
of age and older with Duchenne muscular dystrophy (DMD) that was
discovered through the research and development efforts of
Italfarmaco in collaboration with Telethon and Duchenne Parent
Project (Italy). DUVYZAT is a
histone deacetylase (HDAC) inhibitor that modulates the deregulated
activity of HDACs in the dystrophic muscle, which is a major
consequence of the lack of dystrophin associated with DMD. Though
the exact mechanism of action of DUVYZAT is unknown, it is believed
to inhibit HDAC pathological overactivity to address the cascade of
events leading to muscle damage, thereby counteracting the disease
pathology and slowing down muscle deterioration.
References:
- Duvyzat. Package Insert. ITF Therapeutics, LLC; 2024.
- FDA Approves Nonsteroidal Treatment for Duchenne Muscular
Dystrophy. FDA; March 21, 2024;
https://www.fda.gov/news-events/press-announcements/fda-approves-nonsteroidal-treatment-duchenne-muscular-dystrophy
- Duchenne Muscular Dystrophy. NORD;
https://rarediseases.org/rare-diseases/duchenne-muscular-dystrophy/ (accessed
April 2, 2024)
For more information, please contact
marketing@pantherxrare.com
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SOURCE PANTHERx Rare Pharmacy