Emerging treatments
Peroxisome proliferator-activated receptor (PPAR)-alpha agonists (fibrates)
The PPAR-alpha agonists bezafibrate and fenofibrate have shown significant biochemical benefits in conjunction with ursodiol, although until recently the evidence was low quality.[67]
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The results of a high-quality trial suggest a clear benefit of fibrates in combination with ursodiol, with an acceptable safety profile.[68] Fibrates are recommended as a second-line treatment for patients with PBC who have an inadequate response to ursodiol, although they are discouraged in patients with decompensated liver disease.[14][15][47] Further clarification of their position in the treatment pathway of PBC is needed.
Budesonide
European guidelines recommend budesonide, a corticosteroid that undergoes significant first-pass metabolism, as a second-line treatment for patients with PBC who have an inadequate response to ursodiol.[15] However, the American Association for the Study of Liver Disease suggests that its use remains controversial.[14] Further clarification on the use of budesonide in the treatment pathway of PBC is needed.
Modafinil
Under evaluation for the treatment of fatigue associated with significant daytime somnolence.
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