Mirum Pharmaceuticals has received FDA approval to expand the use of LIVMARLI (maralixibat) oral solution for treating cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC). The label expansion includes patients aged 12 months and older, and the higher concentration formulation was used in the Phase 3 MARCH study.
Chris Peetz, CEO of Mirum, expressed excitement about the availability of LIVMARLI for PFIC patients 12 months and older. Early initiation of treatment is crucial for mitigating the debilitating symptoms associated with this rare liver disease.
LIVMARLI is an oral, once-daily medication that inhibits ileal bile acid transporters (IBAT). It is approved for treating cholestatic pruritus in patients with Alagille syndrome (ALGS) aged three months and older in the US and two months and older in Europe. For PFIC patients, LIVMARLI is approved for use in individuals aged 12 months and older in the US and three months and older in Europe.
Breakthrough Therapy and orphan designations have been granted to LIVMARLI for treating ALGS and PFIC type 2. For more information on ongoing clinical trials, visit Mirum’s website.
LIVMARLI is contraindicated in PFIC type 2 patients with severe defects in the bile salt export pump (BSEP) protein.
Potential side effects of LIVMARLI include:
• Liver injury: Liver function tests should be monitored regularly, and patients should report symptoms of liver problems.
• Gastrointestinal issues: Diarrhea and stomach pain may occur, and patients should seek medical attention if these symptoms worsen or become severe.
• Fat-soluble vitamin deficiency: LIVMARLI can lower levels of certain vitamins, and patients should follow medical advice for monitoring and supplementation.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Ferry, a computer data scientist, focuses on the latest clinical trial industry news and trends.
