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Prescribing Information
  Indication
Important Safety Information
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Always SAFETY First!

>6 Years of Clinical Data
Plus Real-World Use1-3

Backed by >5 years of safety data,* LIVMARLI has a well-characterized safety and tolerability profile for cholestatic pruritus in patients with Alagille syndrome.1,2 In the ICONIC study, there were no discontinuations of LIVMARLI due to ineffectiveness.4

*The majority of exposure occurred without a placebo control in open-label extensions.

Adverse Reactions

Adverse reactions occurring in ≥5% of patients treated with LIVMARLI in the Alagille syndrome clinical development program (n=86)1†

Adverse reactionAny grade n (%)Number of events per
100 person-years§
Diarrhea48 (55.8%)41.6
Abdominal pain‡46 (53.5%)38.6
Vomiting35 (40.7%)19.8
Nausea7 (8.1%)2.9
Fat-soluble vitamin (FSV) deficiency‡22 (25.6%)11.1
Transaminases increased (alanine
aminotransferase [ALT], aspartate
aminotransferase [AST])‡		16 (18.6%)6.9
Gastrointestinal bleeding‡9 (10.4%)3.8
Bone fractures‡8 (9.3%)3.3

†Integrated safety profile from multiple clinical trials, including the ICONIC study.1,2

‡Terms were defined as: FSV deficiency includes vitamins A, D, E, and/or K deficiency, or International Normalized Ratio (INR) increase; abdominal pain includes abdominal discomfort, abdominal distension, abdominal pain, abdominal pain lower, abdominal pain upper; transaminases increased includes ALT abnormal, ALT increased, AST abnormal, AST increased; gastrointestinal bleeding includes hematochezia, hematemesis, gastrointestinal hemorrhage, melena; bone fractures include tibia fracture, rib fracture, humerus fracture, pathogical fracture, forearm fracture, and clavicle fracture.1

§Exposure-adjusted incidence rate for each adverse reaction type was calculated using the first occurrence of this adverse reaction per patient.1

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Additional Safety for LIVMARLI

The most common adverse reactions seen with LIVMARLI in the Alagille syndrome clinical development program, which included 5 clinical studies comprising 86 patients, were diarrhea, abdominal pain, vomiting, FSV deficiency, liver test abnormalities, gastrointestinal bleeding, and bone fractures.1

Five patients experienced treatment interruptions or dose reductions due to diarrhea, abdominal pain, or vomiting.1,5 Among those taking LIVMARLI, no patients discontinued due to diarrhea, abdominal pain, or vomiting.6

Three patients (3%) experienced vomiting as a serious adverse event requiring hospitalization or IV fluid administration.1

In a pooled analysis of patients with Alagille syndrome (n=86), 7 patients discontinued LIVMARLI due to increases in hepatic transaminases (alanine aminotransferase [ALT]) and 3 patients had a decrease in dose or interruption of LIVMARLI in response to these increases; elevations in transaminases were asymptomatic and not associated with bilirubin or other laboratory abnormalities.1

  • In the majority of cases, the elevations resolved or improved after discontinuation or dose modification of LIVMARLI1
  • In some cases, the elevations resolved or improved without change in LIVMARLI dosing1
  • Four patients experienced bilirubin increases above baseline, and LIVMARLI was subsequently withdrawn in 2 of these patients (those who had elevated bilirubin at baseline)1

Patients with Alagille syndrome can have FSV deficiency (vitamins A, D, E, and/or K) at baseline.1

  • LIVMARLI may affect absorption of FSV1

Pediatric patients aged 3 months to <12 months had similar safety, tolerability, and pharmacokinetic profiles to those ≥1 year old.1

Turning Over a New Leaf in Alagille Syndrome

Learn how LIVMARLI—the first FDA-approved treatment for cholestatic pruritus in Alagille syndrome—battles bile acid buildup.1

See How LIVMARLI Works

Encourage patients to download the Itch✓ app to help them track symptom patterns over time and generate customized reports to share at appointments.

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References: 1. LIVMARLI® (maralixibat) oral solution. Prescribing Information. Mirum Pharmaceuticals, Inc. 2. Raman RK, Garner W, Vig P, Tucker E. An integrated analysis of long-term clinical safety in maralixibat-treated participants with Alagille syndrome. Poster presented at: European Association for the Study of the Liver (EASL): International Liver Congress; June 23-26, 2021. 3. Data on file. REF-00349. Mirum Pharmaceuticals, Inc. 4. Gonzales E, Hardikar W, Stormon M, et al. Efficacy and safety of maralixibat treatment in patients with Alagille syndrome and cholestatic pruritus (ICONIC): a randomised phase 2 study. Lancet. 2021;398(10311):1581-1592. doi:10.1016/S0140-6736(21)01256-3 5. Kamath BM, Raman RK, Garner W, Tucker E, Vig P, Gonzales E. Gastrointestinal tolerability of maralixibat in patients with Alagille syndrome: an integrated analysis of short- and long-term treatment. Poster presented at: The 6th World Congress of Pediatric Gastroenterology, Hepatology and Nutrition; June 2-5, 2021; Vienna, Austria. 6. Data on file. REF-00180. Mirum Pharmaceuticals, Inc.

Indication

LIVMARLI is indicated for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 3 months of age and older.

Important Safety Information

Warnings and Precautions

Liver Test Abnormalities: Patients enrolled in clinical trials had abnormal liver tests at baseline. In the main clinical trial, treatment-emergent elevations or worsening of liver tests (ALT, AST or T/DB) relative to baseline were observed. Obtain baseline liver tests and monitor during treatment. Dose reduction or treatment interruption may be considered if abnormalities occur in the absence of other causes. For persistent or recurrent liver test abnormalities, consider treatment discontinuation. Discontinue permanently if a patient progresses to portal hypertension or experiences a hepatic decompensation event.

GI Adverse Reactions: Diarrhea, abdominal pain and vomiting were reported as the most common adverse reactions. If diarrhea, abdominal pain and/or vomiting occur and no other etiologies are found, consider reducing the dose or interrupting LIVMARLI. For diarrhea or vomiting, monitor for dehydration and treat promptly. Consider interrupting LIVMARLI dosing if a patient experiences persistent diarrhea or has diarrhea with accompanying signs and symptoms such as bloody stool, vomiting, dehydration requiring treatment, or fever. Restart LIVMARLI at 190 mcg/kg/day when diarrhea, abdominal pain or vomiting resolve, and increase the dose as tolerated. If they recur upon re-challenge, consider stopping LIVMARLI treatment.

Fat-Soluble Vitamin Deficiency: ALGS patients can have fat-soluble vitamin (FSV) deficiency (vitamins A, D, E, and K) at baseline, and LIVMARLI may affect absorption of FSV. In the main clinical trial, treatment emergent FSV deficiency was reported in 3 (10%) patients during 48 weeks of treatment. Obtain baseline serum levels and monitor during treatment, along with any clinical manifestations. Supplement if deficiency is observed. Consider discontinuing LIVMARLI if FSV deficiency persists or worsens despite adequate FSV supplementation.

Adverse Reactions

The most common adverse reactions (≥5%) are diarrhea, abdominal pain, vomiting, fat-soluble vitamin deficiency, liver test abnormalities, gastrointestinal bleeding 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 30 minutes before a meal in the morning. 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.

Please see full Prescribing Information for LIVMARLI.

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References: 1. LIVMARLI® (maralixibat) oral solution. Prescribing Information. Mirum Pharmaceuticals, Inc. 2. Raman RK, Garner W, Vig P, Tucker E. An integrated analysis of long-term clinical safety in maralixibat-treated participants with Alagille syndrome. Poster presented at: European Association for the Study of the Liver (EASL): International Liver Congress; June 23-26, 2021. 3. Data on file. REF-00349. Mirum Pharmaceuticals, Inc. 4. Gonzales E, Hardikar W, Stormon M, et al. Efficacy and safety of maralixibat treatment in patients with Alagille syndrome and cholestatic pruritus (ICONIC): a randomised phase 2 study. Lancet. 2021;398(10311):1581-1592. doi:10.1016/S0140-6736(21)01256-3 5. Kamath BM, Raman RK, Garner W, Tucker E, Vig P, Gonzales E. Gastrointestinal tolerability of maralixibat in patients with Alagille syndrome: an integrated analysis of short- and long-term treatment. Poster presented at: The 6th World Congress of Pediatric Gastroenterology, Hepatology and Nutrition; June 2-5, 2021; Vienna, Austria. 6. Data on file. REF-00180. Mirum Pharmaceuticals, Inc.

Indication

LIVMARLI is indicated for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 3 months of age and older.

Important Safety Information

Warnings and Precautions

Liver Test Abnormalities: Patients enrolled in clinical trials had abnormal liver tests at baseline. In the main clinical trial, treatment-emergent elevations or worsening of liver tests (ALT, AST or T/DB) relative to baseline were observed. Obtain baseline liver tests and monitor during treatment. Dose reduction or treatment interruption may be considered if abnormalities occur in the absence of other causes. For persistent or recurrent liver test abnormalities, consider treatment discontinuation. Discontinue permanently if a patient progresses to portal hypertension or experiences a hepatic decompensation event.

GI Adverse Reactions: Diarrhea, abdominal pain and vomiting were reported as the most common adverse reactions. If diarrhea, abdominal pain and/or vomiting occur and no other etiologies are found, consider reducing the dose or interrupting LIVMARLI. For diarrhea or vomiting, monitor for dehydration and treat promptly. Consider interrupting LIVMARLI dosing if a patient experiences persistent diarrhea or has diarrhea with accompanying signs and symptoms such as bloody stool, vomiting, dehydration requiring treatment, or fever. Restart LIVMARLI at 190 mcg/kg/day when diarrhea, abdominal pain or vomiting resolve, and increase the dose as tolerated. If they recur upon re-challenge, consider stopping LIVMARLI treatment.

Fat-Soluble Vitamin Deficiency: ALGS patients can have fat-soluble vitamin (FSV) deficiency (vitamins A, D, E, and K) at baseline, and LIVMARLI may affect absorption of FSV. In the main clinical trial, treatment emergent FSV deficiency was reported in 3 (10%) patients during 48 weeks of treatment. Obtain baseline serum levels and monitor during treatment, along with any clinical manifestations. Supplement if deficiency is observed. Consider discontinuing LIVMARLI if FSV deficiency persists or worsens despite adequate FSV supplementation.

Adverse Reactions

The most common adverse reactions (≥5%) are diarrhea, abdominal pain, vomiting, fat-soluble vitamin deficiency, liver test abnormalities, gastrointestinal bleeding 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 30 minutes before a meal in the morning. 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.

Please see full Prescribing Information for LIVMARLI.