Celebrating the repurposed use of Livmarli for pediatric rare liver disease patients
Global Liver Institute (GLI) celebrates a major breakthrough with the U.S. FDA’s approval for Mirum Pharmaceuticals’s Livmarli (maralixibat chloride) to address cholestatic pruritus in patients aged 5 and above with progressive familial intrahepatic cholestasis (PFIC). This repurposed medication offers hope to patients facing limited treatment options, potentially improving their quality of life and longevity. Livmarli, previously approved for Alagille syndrome (ALGS), now extends its benefits to two different rare pediatric liver diseases causing pruritus.
Discover the latest treatment developments in autoimmune hepatitis
Ethan M. Weinberg, MD, MS, assistant professor of clinical medicine at the University of Pennsylvania Perelman School of Medicine, and the Gastroenterology and Hepatology journal shared an insightful Q & A about the treatment landscape for autoimmune hepatitis (AIH) patients. From practice and discussions with other physicians, Dr. Weinberg emphasized the pivotal findings of the CAMARO study’s, suggesting a potential shift in first-line therapy from azathioprine to mycophenolate mofetil and discussed the pressing need for expanded second-line treatment options since almost half of AIH patients in the US do not respond to initial therapies.
Addressing the increase in fatty liver diseases in Latinx children
The prevalence of fatty liver disease is on the rise among children, particularly in the Latinx community, presenting a concerning trend. A recent study conducted by UC San Francisco suggests that this increase may stem from food insecurity and childhood obesity within these communities. Urgent action is needed to enhance screening for nonalcoholic fatty liver disease and prioritize early nutritional interventions.
The U.S. FDA Approves the first gene therapy for rare pediatric disease!
Exciting progress is happening in gene therapy, bringing hope to patients with rare diseases. On March 18, 2024, the FDA approved Orchard Therapeutics’ groundbreaking gene therapy called Lenmeldy (atidarsagene autotemcel). This therapy is a life-saving therapy for children with metachromatic leukodystrophy, a rare condition that causes harmful sulfides to build up in the liver and several other organs, which leads to serious neurological problems over time.
Upcoming Events
- April 6, 2024 – Mid South Liver Alliance’s Living with Liver Disease – Nashville
- April 9 – 12, 2024 – International PBC Summit
- April 23 – 25, 2024 – World Orphan Drug Congress USA 2024
- April 26 – 27, 2024 – PFIC Family & Scientific Conference
- April 29 – May 1, 2024 – Global Genes RARE Drug Development Symposium
- April 11 – 13, 2024 – 2024 Patient Education Conference Empowering Connections in PBC
- May 10 – 11, 2024 – BARE Symposium
For more information about the Pediatric and Rare Liver Diseases Council or to learn more about joining, please visit our webpage or email pedsrare@globalliver.org.
