Advancements in Rare Liver Disease Treatment

Thanks to the dedication of researchers and innovators, patients with several rare liver diseases can find hope in the promise of new treatments on the horizon – great news on the heels of Rare Liver Diseases Month. Join us as we explore the most recent advances in the field and share insights from experts in the rare disease community.

Global Liver Institute Features Resources for Patients and Caregivers

With a focus on pregnancy-related liver diseases, pregnancy in rare liver diseases, and parenthood, GLI launched the 3rd #RareAware Campaign in February 2023 to raise public awareness and education. This campaign featured 4 GLI LIVE episodes, 3 roundtables, and a list of educational resources for patients, providers, and caregivers in the rare liver disease community and more. Stay tuned for the full campaign report!

In Case You Missed It!

View last month’s GLI LIVE episodes:

LIVMARLI, Currently Approved for Pruritus in Alagille Syndrome, to Be Used for Progressive Familial Intrahepatic Cholestasis

LIVMARLI® (maralixibat), a drug that has already been approved for the treatment of pruritus in Alagille syndrome, is now being explored as a treatment option for other rare liver diseases. In a recent announcement, Mirum Pharmaceuticals revealed that they’ve submitted a supplemental New Drug Application for LIVMARLI oral solution to treat cholestatic pruritus in patients two months of age and older with progressive familial intrahepatic cholestasis (PFIC). Data from the MARCH PFIC Phase 3 study have shown significant improvements to pruritus, serum bile acids, bilirubin, and growth in patients with all six forms of PFIC. We look forward to following its progress and potential impact for patients in the coming months.

Olezarsen receives Fast Track Designation for patients with Familial Chylomicronemia Syndrome

Last year, Ionis Pharmaceuticals initiated the Phase 3 BALANCE study on Olezarsen, a medicine for patients who are at risk of disease due to elevated triglyceride levels from the liver. Study data are expected to be available in the second half of 2023. Furthermore, the FDA has granted Olezarsen fast track designation for the treatment of lipoprotein lipase deficiency (LPLD) or familial chylomicronemia syndrome (FCS). New treatment options for patients with these conditions may be right around the corner.

For more information about the Pediatric and Rare Liver Diseases Council or to learn more about joining, please visit https://globalliver.org/pediatric-rare-liver-diseases-council/.

For any questions, reach out to our email: pedsrare@globalliver.org.