New developments in the treatment of primary biliary cholangitis – role of obeticholic acid

Figures & data

Table 1 Summary of major clinical trials of obeticholic acid in primary biliary cholangitis

Authors	Inclusion criteria	Use of UDCA	Duration	Primary endpoints	Number of subjects	OCA dose	Primary endpoints met
Kowdley et al^Citation47	ALKP >1.5× the ULN, No UDCA >6 months	No	12 weeks	% reduction in serum ALKP	59
					23	Placebo	+0.4%
					20	10 mg	45%
					16	50 mg	38%
Hirschfield et al^Citation46	ALKP 1.5–10× the ULN	Yes	12 weeks	% reduction in serum ALKP	165
					38	Placebo	3%
					38	10 mg	24%
					48	25 mg	25%
					41	50 mg	21%
Nevens et al^Citation48	ALKP ≥1.67× the ULN and/or TB more than the ULN to 2× the ULN	Yes	12 months	Serum ALKP of <1.67× the ULN (with >15% reduction from baseline) and normalization of bilirubin	216
					73	Placebo	10%
					70	5–10 mg	46%
					73	10 mg	47%

Abbreviations: ALKP, alkaline phosphatase; OCA, obeticholic acid; TB, total bilirubin; UDCA, ursodeoxycholic acid; ULN, upper limit of normal.

Table 2 OCA clinical trials – absolute change in lipid levels from baseline

	Phase II OCA Kowdley et al^Citation47		Phase II OCA + UDCA Hirschfield et al^Citation46		Phase III OCA ± UDCA Nevens et al^Citation48
	Placebo (n=23)	OCA 10 mg (n=20)	Placebo (n=38)	OCA 10 mg (n=38)	Placebo (n=73)	Titration OCA (n=70)	OCA 10 mg (n=73)
LDL-C (mg/dL)	−3.1	3.9	3.5	9.7	1.4	3.5	−1.9
HDL-C (mg/dL)	−1.5	−12.7	3.5	−9.7	−3.5	−11.2	−16.6
Triglyceride (mg/dL)	−1.8	−2.7	−8.9	−2.7	4.4	−5.3	−14.2

Abbreviations: HDL-C, high-density lipoprotein-cholesterol; LDL-C, low-density lipoprotein-cholesterol; OCA, obeticholic acid; UDCA, ursodeoxycholic acid.

KowdleyKJonesDLuketicVThe OCA PBC Study Group, An international study evaluating the farnesoid X receptor agonist obeticholic acid as monotherapy in PBCJ Hepatol201254S13

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HirschfieldGMMasonALuketicVEfficacy of obeticholic acid in patients with primary biliary cirrhosis and inadequate response to ursodeoxycholic acidGastroenterology20151484751761.e75825500425

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NevensFAndreonePMazzellaGA placebo-controlled trial of obeticholic acid in primary biliary cholangitisN Engl J Med2016375763164327532829

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