FDA Approves Tablet Formulation of LIVMARLI for Rare Pediatric Liver Diseases
核心洞察
Mirum Pharmaceuticals' LIVMARLI (maralixibat) has received FDA approval for a new tablet formulation to treat cholestatic pruritus (搜索) in patients with Alagille syndrome (搜索) and Progressive Familial Intrahepatic Cholestasis (搜索).
The tablet version provides a convenient one-tablet per dose option for older patients, while maintaining the liquid formulation for younger patients, enhancing treatment flexibility for physicians and patients.
LIVMARLI, an ileal bile acid transporter (搜索) (IBAT (搜索)) inhibitor, is now approved in over 40 countries for ALGS (搜索) and 30 countries for PFIC (搜索), with the new tablet formulation expected to be available in June 2025.
Mirum Pharmaceuticals announced today that the U.S. Food and Drug Administration (FDA) has approved a new tablet formulation of LIVMARLI® (maralixibat) for the treatment of cholestatic pruritus (搜索) in patients with Alagille syndrome (搜索) (ALGS (搜索)) and Progressive Familial Intrahepatic Cholestasis (搜索) (PFIC (搜索)).
The tablet formulation provides an additional option alongside the existing oral solution, offering greater flexibility for patients and healthcare providers. The liquid formulation remains available for younger patients, while the new tablet provides a convenient one-tablet per dose option for older individuals.
"The approval of LIVMARLI in tablet form provides a meaningful additional treatment option for patients with ALGS (搜索) and PFIC (搜索)," said Peter Radovich, president and chief operating officer at Mirum. "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."
The company plans to make LIVMARLI tablets available in June 2025 through its Mirum Access Plus program.
Understanding ALGS and PFIC
Alagille syndrome (搜索) and Progressive Familial Intrahepatic Cholestasis (搜索) are rare genetic disorders that cause severe liver dysfunction. Both conditions are characterized by cholestatic pruritus (搜索) – intense, often debilitating itching caused by the buildup of bile acids in the body due to impaired bile flow.
These rare pediatric liver diseases significantly impact quality of life for affected children and their families. Prior to LIVMARLI's approval, treatment options were limited primarily to symptom management or, in severe cases, liver transplantation.
Mechanism of Action and Clinical Applications
LIVMARLI works as an ileal bile acid transporter (搜索) (IBAT (搜索)) inhibitor, reducing bile acid reabsorption in the small intestine and increasing bile acid excretion through the feces. This mechanism helps alleviate cholestatic pruritus (搜索) by reducing the systemic accumulation of bile acids.
The medication is approved for:
- ALGS patients three months of age and older in the U.S. and two months of age and older in Europe
- PFIC patients 12 months of age and older in the U.S. and three months of age and older in Europe
It's important to note that LIVMARLI is not indicated for PFIC (搜索) type 2 patients with severe defects in the bile salt export pump (搜索) (BSEP (搜索)) protein.
Global Expansion and Recent Approvals
The tablet approval follows recent expansion of LIVMARLI's global footprint. In March 2025, Mirum's partner Takeda Pharmaceutical Company (搜索) received approval from the Japanese Ministry of Health, Labour, and Welfare for LIVMARLI oral solution for both ALGS (搜索) and PFIC (搜索) patients.
"We are thrilled to see LIVMARLI approved as the first and only medication available in Japan for patients living with pruritus related to ALGS (搜索) and PFIC (搜索)," said Chris Peetz, chief executive officer at Mirum, regarding the Japanese approval.
LIVMARLI is currently approved for treating pruritus related to ALGS (搜索) in more than 40 countries and for PFIC (搜索) in more than 30 countries worldwide.
Safety Considerations
Healthcare providers should be aware of potential side effects associated with LIVMARLI, including:
- Liver injury: Changes in liver tests are common in ALGS and PFIC patients but may worsen during treatment. In PFIC, this can potentially lead to serious outcomes including liver transplant.
- Gastrointestinal problems: Diarrhea and stomach pain have been reported.
- Fat Soluble Vitamin (FSV) Deficiency: Low levels of vitamins A, D, E, and K may worsen during treatment.
Regular monitoring through blood tests and physical examinations is recommended before and during treatment to assess liver function and vitamin levels.
Ongoing Research
Mirum continues to explore additional applications for LIVMARLI through the Phase 3 EXPAND study, which is evaluating the medication in additional settings of cholestatic pruritus (搜索).
The company's pipeline also includes volixibat, another IBAT (搜索) inhibitor being studied for primary sclerosing cholangitis (搜索) (PSC (搜索)) and primary biliary cholangitis (搜索) (PBC (搜索)). Volixibat has received Breakthrough Therapy Designation for treating cholestatic pruritus (搜索) in PBC patients.
Additionally, Mirum is planning a Phase 2 study of MRM-3379, a PDE4D (搜索) inhibitor, for Fragile X syndrome (搜索), a rare genetic neurocognitive disorder.
The approval of the LIVMARLI tablet formulation represents another step in Mirum's mission to transform treatment options for rare diseases affecting both children and adults.