Mirum Pharmaceuticals has received FDA approval for a tablet formulation of Livmarli (maralixibat), a treatment for skin itching associated with the rare pediatric liver diseases progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome (ALGS).
Livmarli is an ileal bile acid transporter (IBAT) inhibitor used to treat pruritus (intense itching) associated with cholestasis, the slowing of bile flow through the biliary system, in patients with ALGS and PFIC.
The newly approved tablet formulation offers greater flexibility and convenience, especially for older pediatric patients and adolescents transitioning from the previously available oral solution.
“The approval of Livmarli in tablet form provides a meaningful additional treatment option for patients with ALGS and PFIC. It allows flexibility for patients and physicians, with the liquid dosing used by the youngest patients and a convenient one-tablet per dose option for older patients,” said Peter Radovich, president and chief operating officer at Mirum.
“We have had tremendous success with Livmarli since its launch and we hope that the availability of the tablet will provide convenience that positively impacts patients’ lives.”
About ALGS and PFIC
ALGS is a rare genetic disorder that primarily affects the liver, heart and other parts of the body due to abnormalities in bile ducts, which impair bile flow and lead to potential liver damage. Mutations in the JAG1 or NOTCH2 genes, which play a key role in the development of bile ducts and other tissues, are the underlying cause of the disease.
Patients with the condition often develop cholestasis, severe itching, heart defects, distinct facial features and skeletal or eye abnormalities.
PFIC is a group of rare, inherited liver disorders that cause bile to build up in the liver due to defects in bile secretion. This buildup leads to cholestasis, resulting in symptoms such as intense itching (pruritus), jaundice, growth problems and eventually liver damage or failure.
PFIC typically appears in infancy or early childhood and is caused by mutations in genes like ATP8B1, ABCB11 or ABCB4 that affect bile transport. Without treatment, many patients may require a liver transplant at a young age.
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Livmarli and Similar Therapies
Livmarli works by blocking IBAT, reducing the build-up of bile acids that contribute to severe itching and liver damage in ALGS and PFIC.
The FDA initially approved the drug in an oral solution form in 2021 for ALGS. The agency then granted it expanded approval to include PFIC in 2024.
The new tablet version will allow more personalized dosing options and may enhance adherence to long-term therapy — an important consideration in managing chronic pediatric conditions.
Mirum said it plans for Livmarli tablets to be available in June. The company will offer them through its patient support program Mirium Access Plus.
Related: Ctexli (Chenodiol) Gains FDA Approval as First CTX Treatment
The company is also continuing to evaluate Livmarli for pruritus in other rare cholestatic settings in ongoing clinical trials.
Earlier this year, Mirum won FDA approval for its bile acid tablets Ctexli (chenodiol) for the treatment of the rare bile acid metabolism disorder cerebrotendinous xanthomatosis (CTX).
The FDA approved Albireo Pharma’s (now part of Ipsen) IBAT inhibitor Bylvay (odevixibat) in July 2021 for pruritus in PFIC. It went on to secure an approval in ALGS in 2023.
In 2024, Gilead won approval for Livdelzi (seladelpar), a selective peroxisome proliferator-activated receptor delta (PPARδ) agonist, for primary biliary cholangitis (PBC). Gilead received the drug in its acquisition of CymaBay Therapeutics earlier that year in a $4.3 billion deal.
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