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UNDERSTANDING SAFETY IN OUR NECK OF THE WOODS

Well-Characterized Safety and Tolerability Profile

Adverse Reactions

LIVMARLI Has Well-Established Safety and Tolerability for Cholestatic Pruritus in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)1,2

Adverse reactions occurring in ≥5% and at a rate greater than placebo in patients treated with LIVMARLI in the MARCH-PFIC clinical study1
Adverse ReactionLIVMARLI (n=47)
n (%)		Placebo (n=46)
n (%)
Diarrhea27 (57.4%)9 (19.6%)
Abdominal pain*13 (27.7%)7 (15.2%)
Transaminases increased (ALT or AST)*8 (17%)3 (6.5%)
Hematochezia or rectal hemorrhage*4 (8.5%)1 (2.2%)
Bone fractures*3 (6.4%)0

ALT=alanine transaminase; AST=aspartate aminotransferase.

*Abdominal pain includes abdominal pain, abdominal pain upper, and abdominal distension; transaminases increased includes hypertransaminasaemia, ALT abnormal, ALT increased, AST abnormal, AST increased, transaminases increased, and hepatic enzyme increased; bone fractures include upper limb fracture, lower limb fracture, radius fracture, ulna fracture, femur fracture, and foot fracture.

bird on branch bird on branch

Additional Safety for LIVMARLI

The most common adverse reaction seen with LIVMARLI in the MARCH-PFIC study was diarrhea.1

Diarrhea was the most frequent adverse reaction; the majority of episodes were mild and transient. Diarrhea resolved, on average, within 5.5 days.1,2

  • One patient with an event of mild diarrhea discontinued treatment1,2
  • Treatment interruptions or dose reductions occurred in 3 patients due to diarrhea or abdominal pain1

Treatment with LIVMARLI is associated with a potential for drug-induced liver injury.1

  • Treatment-emergent elevations of liver tests or worsening of liver tests, relative to baseline values, and hepatic decompensation events were observed1
  • Two patients experienced drug-induced liver injury (DILI) attributable to LIVMARLI. One patient received 570 mcg/kg twice daily and the second patient required dose interruption and reduction1
  • Two additional patients experienced DILI in the open-label extension portion of the trial1
  • Of these 4 patients, 1 patient required liver transplant and another patient died. The contribution of LIVMARLI in these 2 cases is uncertain1
  • In the placebo-controlled arm of the trial, 2 patients treated with LIVMARLI developed cholangitis or cholecystitis within 3 weeks of drug discontinuation. Four patients treated with LIVMARLI developed cholecystitis or cholangitis in the open-label extension1
  • Treatment-emergent bone fracture events were observed. Three patients treated with LIVMARLI experienced bone fractures compared with none with placebo. Two patients treated with LIVMARLI developed bone fractures in the open-label extension1
  • Patients receiving LIVMARLI reported more frequent treatment-emergent events of hematochezia (4 [8.5%] vs 1 [2.2%]), and a decrease in hemoglobin ≥2 grams/dL from baseline (8 [17%] vs 1 [2.2%]) compared with patients receiving placebo1

Patients with PFIC can have fat-soluble vitamin (FSV) deficiency (vitamins A, D, E, and/or K) at baseline.1

  • LIVMARLI may affect absorption of FSV1
  • In patients with PFIC, treatment-emergent FSV deficiency was reported in 13 (28%) patients treated with LIVMARLI vs 16 (35%) patients treated with placebo during 26 weeks of treatment1

Improvements in Cholestatic Pruritus

Meaningful improvements in cholestatic pruritus were seen as early as 2 weeks and sustained through 2 years.1-3

See the Efficacy Data

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References: 1. LIVMARLI® (maralixibat) oral solution. Prescribing Information. Mirum Pharmaceuticals, Inc. 2. Miethke AG, Moukarzel A, Porta G, et al. Maralixibat in progressive familial intrahepatic cholestasis (MARCH-PFIC): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Gastroenterol Hepatol. 2024;9(7):620-631. doi:10.1016/s2468-1253(24)00080-3 3. Miethke A, Moukarzel A, Porta G, et al. Long-term maintenance of response and improved liver health with maralixibat in patients with progressive familial intrahepatic cholestasis (PFIC): 2-year data from the MARCH-ON study. Presented at: American Association for the Study of Liver Diseases (AASLD) The Liver Meeting: November 10-14, 2023; Boston, MA.

INDICATION

LIVMARLI is indicated for the treatment of cholestatic pruritus in patients 12 months of age and older with progressive familial intrahepatic cholestasis (PFIC).

Limitations of Use: LIVMARLI is not recommended in a subgroup of PFIC type 2 patients with specific ABCB11 variants resulting in nonfunctional or complete absence of bile salt export pump (BSEP) protein.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

LIVMARLI is contraindicated in patients with prior or active hepatic decompensation events (eg, variceal hemorrhage, ascites, or hepatic encephalopathy).

WARNINGS AND PRECAUTIONS

Hepatotoxicity: LIVMARLI treatment is associated with a potential for drug-induced liver injury (DILI).

In the PFIC trial, treatment-emergent hepatic decompensation events and elevations of liver tests or worsening of liver tests occurred. Two patients experienced DILI attributable to LIVMARLI. Two additional patients experienced DILI in the open-label extension portion of the trial. Of these 4 patients, 1 patient required liver transplant and another patient died.

Obtain baseline liver tests and monitor during treatment. Liver-related adverse reactions and physical signs of hepatic decompensation should also be monitored. Dose reduction or treatment interruption may be considered if abnormalities occur in the absence of other causes. Permanently discontinue LIVMARLI if a patient experiences the following: persistent or recurrent liver test abnormalities, clinical hepatitis upon rechallenge, or a hepatic decompensation event.

Gastrointestinal (GI) Adverse Reactions: Diarrhea and abdominal pain were reported as the most common adverse reactions. Monitor for dehydration and treat promptly. Consider reducing the dosage or interrupting LIVMARLI dosing if a patient experiences persistent diarrhea or diarrhea with bloody stool, vomiting, dehydration requiring treatment, or fever.

Fat-Soluble Vitamin (FSV) Deficiency: Patients can have FSV deficiency (vitamins A, D, E, and K) at baseline, and LIVMARLI may adversely affect absorption of FSVs. Treatment-emergent bone fracture events have been observed more frequently with patients treated with LIVMARLI compared with patients treated with placebo. If bone fractures or bleeding occur, consider interrupting LIVMARLI and supplement with FSVs. LIVMARLI can be restarted once FSV deficiency is corrected and maintained at corrected levels.

Risk of Propylene Glycol Toxicity (Pediatric Patients Less Than 5 Years of Age): Total daily intake of propylene glycol should be considered for managing the risk of propylene glycol toxicity. Monitor patients for signs of propylene glycol toxicity. Discontinue LIVMARLI if toxicity is suspected.

ADVERSE REACTIONS

The most common adverse reactions are diarrhea, FSV deficiency, abdominal pain, liver test abnormalities, hematochezia, and bone fractures.

DRUG INTERACTIONS

Administer bile acid binding resins at least 4 hours before or 4 hours after administration of LIVMARLI.
A decrease in the absorption of OATP2B1 substrates (eg, statins) due to OATP2B1 inhibition by LIVMARLI in the GI tract cannot be ruled out. Consider monitoring the drug effects of OATP2B1 substrates as needed.

DOSING INFORMATION

LIVMARLI should be taken twice daily 30 minutes before a meal. The provided oral dosing dispenser must be used to accurately measure the dose. Any remaining LIVMARLI should be discarded 100 days after first opening the bottle.

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References: 1. LIVMARLI® (maralixibat) oral solution. Prescribing Information. Mirum Pharmaceuticals, Inc. 2. Miethke AG, Moukarzel A, Porta G, et al. Maralixibat in progressive familial intrahepatic cholestasis (MARCH-PFIC): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Gastroenterol Hepatol. 2024;9(7):620-631. doi:10.1016/s2468-1253(24)00080-3 3. Miethke A, Moukarzel A, Porta G, et al. Long-term maintenance of response and improved liver health with maralixibat in patients with progressive familial intrahepatic cholestasis (PFIC): 2-year data from the MARCH-ON study. Presented at: American Association for the Study of Liver Diseases (AASLD) The Liver Meeting: November 10-14, 2023; Boston, MA.

INDICATION

LIVMARLI is indicated for the treatment of cholestatic pruritus in patients 12 months of age and older with progressive familial intrahepatic cholestasis (PFIC).

Limitations of Use: LIVMARLI is not recommended in a subgroup of PFIC type 2 patients with specific ABCB11 variants resulting in nonfunctional or complete absence of bile salt export pump (BSEP) protein.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

LIVMARLI is contraindicated in patients with prior or active hepatic decompensation events (eg, variceal hemorrhage, ascites, or hepatic encephalopathy).

WARNINGS AND PRECAUTIONS

Hepatotoxicity: LIVMARLI treatment is associated with a potential for drug-induced liver injury (DILI).

In the PFIC trial, treatment-emergent hepatic decompensation events and elevations of liver tests or worsening of liver tests occurred. Two patients experienced DILI attributable to LIVMARLI. Two additional patients experienced DILI in the open-label extension portion of the trial. Of these 4 patients, 1 patient required liver transplant and another patient died.

Obtain baseline liver tests and monitor during treatment. Liver-related adverse reactions and physical signs of hepatic decompensation should also be monitored. Dose reduction or treatment interruption may be considered if abnormalities occur in the absence of other causes. Permanently discontinue LIVMARLI if a patient experiences the following: persistent or recurrent liver test abnormalities, clinical hepatitis upon rechallenge, or a hepatic decompensation event.

Gastrointestinal (GI) Adverse Reactions: Diarrhea and abdominal pain were reported as the most common adverse reactions. Monitor for dehydration and treat promptly. Consider reducing the dosage or interrupting LIVMARLI dosing if a patient experiences persistent diarrhea or diarrhea with bloody stool, vomiting, dehydration requiring treatment, or fever.

Fat-Soluble Vitamin (FSV) Deficiency: Patients can have FSV deficiency (vitamins A, D, E, and K) at baseline, and LIVMARLI may adversely affect absorption of FSVs. Treatment-emergent bone fracture events have been observed more frequently with patients treated with LIVMARLI compared with patients treated with placebo. If bone fractures or bleeding occur, consider interrupting LIVMARLI and supplement with FSVs. LIVMARLI can be restarted once FSV deficiency is corrected and maintained at corrected levels.

Risk of Propylene Glycol Toxicity (Pediatric Patients Less Than 5 Years of Age): Total daily intake of propylene glycol should be considered for managing the risk of propylene glycol toxicity. Monitor patients for signs of propylene glycol toxicity. Discontinue LIVMARLI if toxicity is suspected.

ADVERSE REACTIONS

The most common adverse reactions are diarrhea, FSV deficiency, abdominal pain, liver test abnormalities, hematochezia, and bone fractures.

DRUG INTERACTIONS

Administer bile acid binding resins at least 4 hours before or 4 hours after administration of LIVMARLI.
A decrease in the absorption of OATP2B1 substrates (eg, statins) due to OATP2B1 inhibition by LIVMARLI in the GI tract cannot be ruled out. Consider monitoring the drug effects of OATP2B1 substrates as needed.

DOSING INFORMATION

LIVMARLI should be taken twice daily 30 minutes before a meal. The provided oral dosing dispenser must be used to accurately measure the dose. Any remaining LIVMARLI should be discarded 100 days after first opening the bottle.