PITTSBURGH, Aug. 16, 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 Gilead Sciences, Inc. for the distribution of a novel selective peroxisome proliferator-activated receptor delta (PPARδ) agonist to treat adults with primary biliary cholangitis (PBC).
LIVDELZI is indicated for the treatment of primary biliary cholangitis (PBC), in combination with ursodeoxycholic acid (UDCA) in adults who have had an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA. This indication is approved under accelerated approval based on a reduction of alkaline phosphatase. LIVDELZI is a first-in-class PPARδ agonist treatment option for patients with PBC.
Primary biliary cholangitis (PBC) is a rare, chronic, and progressive disease of the small bile ducts in the liver. Damage to the small bile ducts causes impaired bile flow, known as cholestasis, and results in the accumulation of toxic bile acids in the liver leading to further inflammation, injury, and cirrhosis. PBC affects mostly women of middle age but can also occur in men. The most common and often debilitating symptoms include fatigue and intense pruritus, which can both negatively impact quality of life. Key goals of treatment include ALP normalization and improvement of PBC-associated itching (pruritis). If PBC is untreated or there is an incomplete response to medical treatment, progressive damage can lead to liver failure.
“People living with rare and devastating diseases deserve access to treatment options that work for them. The partnership with Gilead Sciences aims to positively impact key clinical outcomes for those suffering from Primary Biliary Cholangitis and we are pleased to be a part in that,” said Rob Snyder, Executive Chair 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.
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SOURCE PANTHERx Rare Pharmacy
