Table 6.

Medications under evaluation in randomized, placebo-controlled trials for the treatment of PSC

Drug	Mechanism of action	Phase II results
norUDCA	induction of bicarbonate-rich choleresis, biliary HCO3− “umbrella” strengthening	dose-dependent reduction of ALP, GGT, AST and ALT
Obeticholic acid	FXR agonism (steroidal)	dose-dependent reduction of ALP, no change in fibrosis markers
Cilofexor	FXR agonism (non-steroidal)	dose-dependent improvement of ALP, transaminases and markers of fibrosis
Aldafermin	FGF19 analogue: down-regulation of bile acid synthesis	reduction of serum transaminases and fibrogenesis markers, no effect on ALP
Bezafibrate	PPAR agonism: reduction of bile acid synthesis and increased phospholipid secretion	reduction of ALP improvement of pruritus
Vancomycin	modulation of the intestinal microbiota, anti-inflammatory effects	ALP reduction, Mayo Risk Score decline, symptoms decrease