Mirum Pharmaceuticals will participate in the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition (NASPGHAN) annual meeting from November 6-9, 2024, in Hollywood, Florida. The conference will feature presentations on clinical benefits and real-world evidence for LIVMARLI (maralixibat) in treating Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC).
Several abstracts have been accepted for presentation and are available in the NASPGHAN Program Book. Full analyses will be published on Mirum’s website after the presentations. Key presentations include data on maralixibat’s impact on growth in PFIC patients from the MARCH/MARCH-ON trials, presented by Dr. Amal Aqul. Dr. Karen Murray will present findings from the MERGE study demonstrating the durable clinical benefits of maralixibat for ALGS patients over seven years of treatment. Dr. Alexander Miethke will present two abstracts: one exploring the correlation between improved serum bile acids and liver health markers in PFIC patients treated with maralixibat, and another highlighting the association between pruritus improvement and enhanced quality of life in patients with cholestatic liver disease treated with the drug. Finally, Dr. Jolan Terner-Rosenthal will present real-world data from the United States on the long-term effects of maralixibat on concomitant medication use for cholestatic pruritus in ALGS.
LIVMARLI, an ileal bile acid transporter (IBAT) inhibitor, is approved for treating cholestatic pruritus in ALGS patients three months and older in the U.S. and two months and older in Europe. It’s also approved for treating cholestatic pruritus in PFIC patients 12 months and older in the U.S. and three months and older in Europe. It is important to note that LIVMARLI is unsuitable for PFIC type 2 patients with severe bile salt export pump (BSEP) protein defects.
Potential side effects of LIVMARLI include liver injury, though changes in liver tests are common in ALGS and PFIC patients. Gastrointestinal issues like diarrhea and stomach pain can also occur. Patients may also experience worsened Fat Soluble Vitamin (FSV) Deficiency, a common condition in ALGS and PFIC. Regular monitoring and communication with healthcare providers are crucial for managing these potential side effects. Further information for U.S. residents can be found at LIVMARLI.com. LIVMARLI holds a Breakthrough Therapy designation for both ALGS and PFIC type 2 and an Orphan Drug designation for both conditions.
Mirum Pharmaceuticals focuses on transforming rare disease treatment in children and adults. Besides LIVMARLI, Mirum also markets CHOLBAM (cholic acid) for bile acid synthesis disorders and peroxisomal disorders and CHENODAL (chenodiol) for cerebrotendinous xanthomatosis (CTX). Their pipeline includes volixibat, an IBAT inhibitor being studied for primary sclerosing cholangitis (PSC), primary biliary cholangitis (PBC), and chenodiol, currently under FDA review for CTX treatment.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
