FDA Expands Livmarli Approval for PFIC Patients Aged 12 Months and Older
Key Insights
The FDA has approved Mirum Pharmaceuticals' Livmarli oral solution for treating cholestatic pruritus (search) in PFIC patients aged 12 months and older.
This label expansion includes the higher concentration formulation of Livmarli, which was evaluated in the Phase 3 MARCH study.
Livmarli, an ileal bile acid transporter (IBAT (search)) inhibitor, is already approved for Alagille syndrome (search) and PFIC in other age groups and regions.
Mirum Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has approved a label expansion for Livmarli (maralixibat) oral solution, allowing its use for the treatment of cholestatic pruritus (search) in patients with progressive familial intrahepatic cholestasis (search) (PFIC) aged 12 months and older. This approval extends the availability of Livmarli to a younger patient population, potentially mitigating the long-term effects of pruritus associated with this rare liver disease.
The expanded label includes the higher concentration formulation of Livmarli, which was assessed in the MARCH Phase 3 study. Livmarli functions as an orally administered, once-daily ileal bile acid transporter (IBAT (search)) inhibitor.
"The launch of Livmarli in PFIC is going well and we are thrilled that it will now be available for patients 12 months and older," said Chris Peetz, chief executive officer at Mirum. "PFIC is generally diagnosed when children are young, and initiating treatment quickly after diagnosis will help to ensure they have fewer days suffering from pruritus associated with this rare liver disease."
Livmarli is also approved for cholestatic pruritus (search) in patients with Alagille syndrome (search) (ALGS) aged three months and older in the U.S., and two months and older in Europe. In the U.S., it was previously approved for PFIC patients 12 months and older, while in Europe, it is approved for PFIC patients three months of age and older.
Safety Information
It is important to note that Livmarli is not indicated for PFIC type 2 patients with a severe defect in the bile salt export pump (BSEP) protein.
Adverse effects associated with Livmarli include potential liver injury, as indicated by changes in liver function tests. Patients with Alagille syndrome (search) and PFIC may experience worsening of these tests during treatment, which could signal liver injury, potentially leading to liver transplant or death in PFIC patients. Regular monitoring of liver function is recommended. Patients should immediately report any signs or symptoms of liver problems, such as nausea, vomiting, jaundice, dark urine, abdominal pain, bloating, loss of appetite, or easy bleeding/bruising.
Additionally, Livmarli can cause gastrointestinal issues, including diarrhea and stomach pain. Monitoring for new or worsening stomach problems, such as blood in the stool or vomiting, is advised. Fat-soluble vitamin (FSV) deficiencies are common in patients with Alagille syndrome (search) and PFIC and may worsen during Livmarli treatment. Monitoring for FSV deficiencies, bone fractures, and bleeding is recommended.
Mirum Pharmaceuticals' Pipeline
Mirum Pharmaceuticals is focused on developing treatments for rare liver diseases. Their portfolio includes Livmarli, CHOLBAM (cholic acid) capsules, and CHENODAL (chenodiol) tablets. Volixibat, another IBAT (search) inhibitor, is currently under evaluation for primary sclerosing cholangitis (search) (PSC) and primary biliary cholangitis (search). Chenodiol has also shown positive results in a Phase 3 clinical study for cerebrotendinous xanthomatosis (search) (CTX), and a new drug application has been submitted to the FDA for its approval in treating CTX in the U.S.