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Primary biliary cholangitis (PBC) is an acquired, immune-mediated cholestatic liver disease. The condition is classified as multifactorial autoimmune rather than Mendelian, with no causative genes or Mendelian inheritance patterns documented in this packet. Five disease subtypes are listed in this packet — designated PBC 1 through PBC 5 — representing genetic susceptibility loci identified through genomic association studies rather than distinct clinical phenotypic variants.
Phenotypic data, characteristic clinical findings, and diagnostic methods are not established in this packet's structured fields.
PBC is an acquired autoimmune condition. No causative genes, molecular mechanism data, or Mendelian inheritance patterns are documented in this packet, consistent with its multifactorial immunological etiology.
Specific diagnostic criteria and structured diagnostic methods are not established in this packet's fields.
Three FDA-approved therapies for PBC carry active market status in this packet. Ursodiol (URSO 250) received NDA approval on December 10, 1997, and is currently marketed with active status. Elafibranor (IQIRVO) received NDA approval on June 10, 2024, and is currently marketed with active status. Seladelpar lysine (LIVDELZI) received NDA approval on August 14, 2024, and is currently marketed with active status.
62 trials found
Prognostic information is not established in this packet's structured fields.
Three clinical trials are documented in this packet. NCT04526665 is a Phase 3 study of elafibranor in patients with PBC, sponsored by Ipsen, currently active but not recruiting, with planned completion December 2028. NCT06016842 is a Phase 3 long-term study of elafibranor also sponsored by Ipsen, currently recruiting, with planned completion May 2029. NCT07795151 is a Phase 1 to Phase 2 trial investigating human umbilical cord mesenchymal stromal cells for UDCA-refractory PBC, sponsored by Beijing 302 Hospital, not yet recruiting as of this packet, with planned initiation August 2026 and planned completion August 2030.
Data assembled from 5 of 12 sources · Last updated Nov 2, 2026, 9:06 PM UTC
Patient Advocacy Groups (PAGs) provide support, resources, and community for patients and caregivers.
European rare disease database
Genetic and Rare Diseases Info Center
AI-curated news mentioning primary biliary cholangitis
Updated Oct 6, 2026
Interim analysis from the ASSURE trial shows that seladelpar significantly improves liver stiffness in patients with primary biliary cholangitis (PBC). This finding supports the potential of seladelpar as a therapeutic option for this rare liver disease.
Recent research indicates that statins may reduce the risk of disease progression and mortality in non-cirrhotic patients with primary biliary cholangitis. This finding could influence treatment strategies for managing this rare liver disease.
A systematic review and meta-analysis evaluated the safety profile of elafibranor in patients with primary biliary cholangitis compared to placebo. The findings contribute to understanding the drug's safety in this rare liver disease.
The Scottish Medicines Consortium has accepted Livdelzi for the treatment of primary biliary cholangitis, enhancing treatment options for patients suffering from chronic itch associated with the disease. This decision marks a significant step in addressing the unmet needs of this patient population.
The CHMP has recommended Lynavoy for EU approval as the first IBAT inhibitor targeting cholestatic pruritus in primary biliary cholangitis (PBC). This marks a significant advancement in treatment options for patients suffering from this rare condition.