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Disease

Progressive familial intrahepatic cholestasis type 1

Also known as Byler disease, FIC1 deficiency, PFIC1, cholestasis, progressive familial intrahepatic 1+6 more

Byler disease, FIC1 deficiency, PFIC1, cholestasis, progressive familial intrahepatic 1, cholestasis, progressive familial intrahepatic, type 1, Byler's disease, cholestasis, fatal intrahepatic, cholestasis, progressive familial intrahepatic, 1, progressive familial intrahepatic cholestasis, severe ATP8B1 deficiency.

5
Associated genes
1
Related proteins

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

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Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies

Approval — Livmarli is indicated for the treatment of: Cholestatic pruritus in patients with Alagil… (2022)

Odevixibatapproved

Accelerated approval — Bylvay is indicated for the treatment of progressive familial intrahepatic cholestasis (P… (2021)

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals
2022emaApprovalMaralixibat chloride· Livmarli is indicated for the treatment of: Cholestatic pruritus in patients with Alagille syndrome (ALGS) 2 months of age and older, Progressive familial intrahepatic cholestasis (PFIC) in patients 3 months of age and older. Livmarli tablets is indicated in adults and adolescents 12 years and older for the treatment of: Cholestatic pruritus in patients with Alagille syndrome (ALGS), Progressive familial intrahepatic cholestasis (PFIC). source ↗
2021emaAccelerated approvalOdevixibat· Bylvay is indicated for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months or older (see sections 4.4 and 5.1). source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Associated genes

5 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

Disease biology

1 match

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Reference

Authoritative identity, definition & identifiers.

Synonyms

Byler disease, FIC1 deficiency, PFIC1, cholestasis, progressive familial intrahepatic 1, cholestasis, progressive familial intrahepatic, type 1, Byler's disease, cholestasis, fatal intrahepatic, cholestasis, progressive familial intrahepatic, 1, progressive familial intrahepatic cholestasis, severe ATP8B1 deficiency

References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.