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.