Efficacy and safety of maralixibat treatment in patients with Alagille syndrome and cholestatic pruritus (ICONIC): a randomised phase 2 study
Study (n=31) found the sodium-dependent, bile acid transport inhibitor was associated with clinically meaningful improvements in cholestasis (–117 μmol/L [95% CI –232 to –2] least square mean difference in serum bile acid from end of treatment to end of withdrawal phase).
Source:
The Lancet
SPS commentary:
Alagille syndrome is a cholestatic liver disease that leads to pruritus, xanthomas, and impaired quality of life, for which there are no approved medical therapies. Because intrahepatic bile acids are thought to be the main driver behind hepatocellular injury in cholestatic diseases, pharmacological inhibition of the apical sodium-dependent bile acid transporter has the potential to improve pruritus and disease trajectory.