Pharmacological Breakthroughs: The Role of IBAT Inhibitors
The most significant change in ALGS care by 2026 is the widespread use of Ileal Bile Acid Transporter (IBAT) inhibitors, such as Maralixibat and Odevixibat. Before these drugs, the "intractable" itching (pruritus) associated with the syndrome was often so severe that children would scratch until they bled, frequently requiring a liver transplant solely to stop the itch.
IBAT inhibitors work by blocking the reabsorption of bile acids in the small intestine, forcing them to be excreted. Recent 2026 data shows that long-term use (over 5 years) not only improves quality of life by reducing itching but may also stabilize liver function and reduce the growth of xanthomas (cholesterol deposits under the skin). This has significantly lowered the "transplant rate" for ALGS patients who do not have full liver failure.