PANTHERx®Rare Partnering with ITF Therapeutics LLC for U.S. Distribution of DUVYZAT™

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:

1. Duvyzat. Package Insert. ITF Therapeutics, LLC; 2024.
2. 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
3. Duchenne Muscular Dystrophy. NORD; https://rarediseases.org/rare-diseases/duchenne-muscular-dystrophy/ (accessed April 2, 2024)

For more information, please contact [email protected]

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SOURCE PANTHERx Rare Pharmacy