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IDENTIFYING PATIENTS

Your patients’ outcomes start with your treatment decision.

In Alagille syndrome, it’s important to diagnose early and accurately.1 To do so, look for clinical features from 3 of the following 7 clinical criteria.2*†

Hepatic criteria
HEPATIC
  • Cholestasis
  • Jaundice
  • Hepatomegaly
Cardiac criteria
CARDIAC
  • Pulmonary stenosis
  • Tetralogy of Fallot
Facial criteria
FACIAL
  • Prominent forehead
  • Pointed chin
  • Deep‐set eyes
  • Bulbous tipped nose
Ocular criteria
OCULAR
  • Posterior embryotoxon
  • Optic disk drusen
Skeletal criteria
SKELETAL
  • Butterfly vertebrae
  • Pathological fractures
Renal criteria
RENAL
  • Renal dysplasia
  • Renal tubular acidosis
Vascular criteria
VASCULAR
  • Intracranial bleeding
  • Central nervous system (CNS) vascular malformations

*Absent of a molecular diagnosis or family history.

†Does not represent all possible clinical features.

From Diagnosis to Patient Identification

5-Year-Old Patient

Living With a Bothersome Itch That Won’t Quit

Medical History

  • Diagnosis: Diagnosed with Alagille syndrome at 1 year old, following identification of 4 (of 7) clinical criteria: jaundice, mild hepatomegaly, renal dysplasia, and posterior embryotoxon
    • Confirmatory genetic testing revealed a mutation in NOTCH2
  • Surgical interventions: None
  • Prior medication: Topical steroids
  • Current medication: Ursodiol

Physical Examination

  • Cholestatic pruritus:
    • CSS: 1 (rubbing or mild scratching when undistracted)
    • ItchRO(Obs) score: 2 (moderate itch)
  • Isolated patches of skin are red and inflamed from scratching
  • Despite current treatment, patient’s mother notes that scratching is persistent and with a diurnal pattern, occurring most often in the morning

Lab Results

  • sBA: 250 μmol/L (reference range: 0.0 to 10.0 µmol/L3,4)
5-year-old patient

Do you see patients like this in your practice?

Find out if LIVMARLI could help

7-Year-Old Patient

Still Scratching Despite Current Treatment

Medical History

  • Diagnosis: Diagnosed with Alagille syndrome at 6 months old, following identification of 4 (of 7) clinical criteria: jaundice, hepatomegaly, characteristic facial features, and butterfly vertebrae
    • Confirmatory genetic testing revealed a mutation in JAG1
    • Initially misdiagnosed as biliary atresia
  • Surgical interventions: Kasai procedure
  • Prior medication: Antihistamines
  • Current medication: Ursodiol

Physical Examination

  • Cholestatic pruritus:
    • CSS: 2 (active scratching without abrasions)
    • ItchRO(Pt) score: 2 (moderate itch)
  • Large areas of inflamed skin
  • Scars from old scratch wounds
  • Sleep disturbances
  • Patient’s mother is concerned that continued scratching will lead to open wounds
  • Often wears long-sleeved clothing to hide scratches and wounds

Lab Results

  • sBA: 325 μmol/L (reference range: 0.0 to 10.0 µmol/L3,4)
7-year-old patient

Do you see patients like this in your practice?

Find out if LIVMARLI could help

18-Month-Old Patient

Suffering From Abrasions and Bleeding Despite SOC Treatments

Medical History

  • Diagnosis: Diagnosed with Alagille syndrome at 6 months old, following identification of 3 (of 7) clinical criteria: pulmonary stenosis, posterior embryotoxon, and jaundice
    • Confirmatory genetic testing revealed a mutation in JAG1
  • Surgical interventions: None
  • Prior medication: Antihistamines, topical steroids
  • Current medication: Ursodiol, rifampicin

Physical Examination

  • Cholestatic pruritus:
    • CSS: 4 (cutaneous mutilations, hemorrhage, scarring)
    • ItchRO(Obs) score: 3 (severe itch)
  • Fussiness and irritability
  • Significant sleep disturbances
  • Numerous abrasions and large areas of red, inflamed skin
  • Patient’s father notes that bedsheets are regularly bloodied due to open wounds from scratching

Lab Results

  • sBA: 350 μmol/L (reference range: 0.0 to 10.0 µmol/L3,4)
18-month-old patient

Do you see patients like this in your practice?

Find out if LIVMARLI could help

Get Your Patients Started

Take a closer look at who may be appropriate for LIVMARLI.

Download the Patient Profiles

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.5

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.

Check Out the Itch✓ App

Mirum Access Plus assists both you and your patients at every turn, helping you navigate the payer approval process—and beyond—with ease.

Learn More About
Mirum Access Plus

CSS=Clinician Scratch Scale; ItchRO(Obs)=Itch Reported Outcome (Observer); ItchRO(Pt)=Itch Reported Outcome (Patient); JAG1=jagged canonical Notch ligand 1; NOTCH2=neurogenic locus notch homolog protein 2; sBA=serum bile acid; SOC=standard of care.

References: 1. Kamath BM, Baker A, Houwen R, Todorova L, Kerkar N. Systematic review: the epidemiology, natural history, and burden of Alagille syndrome. J Pediatr Gastroenterol Nutr. 2018;67(2):148-156. doi:10.1097/MPG.0000000000001958 2. Ayoub MD, Kamath BM. Alagille syndrome: diagnostic challenges and advances in management. Diagnostics (Basel). 2020;10(11):907. doi:10.3390/diagnostics10110907 3. Bile acids. Labcorp.com. Accessed May 23, 2023. https://www.labcorp.com/tests/010330/bile-acids 4. Jahnel J, Zöhrer E, Scharnagl H, Erwa W, Fauler G, Stojakovic T. Reference ranges of serum bile acids in children and adolescents. Clin Chem Lab Med. 2015;53(11):1807-1813. doi:10.1515/cclm-2014-1273 5. LIVMARLI® (maralixibat) oral solution. Prescribing Information. 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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CSS=Clinician Scratch Scale; ItchRO(Obs)=Itch Reported Outcome (Observer); ItchRO(Pt)=Itch Reported Outcome (Patient); JAG1=jagged canonical Notch ligand 1; NOTCH2=neurogenic locus notch homolog protein 2; sBA=serum bile acid; SOC=standard of care.

References: 1. Kamath BM, Baker A, Houwen R, Todorova L, Kerkar N. Systematic review: the epidemiology, natural history, and burden of Alagille syndrome. J Pediatr Gastroenterol Nutr. 2018;67(2):148-156. doi:10.1097/MPG.0000000000001958 2. Ayoub MD, Kamath BM. Alagille syndrome: diagnostic challenges and advances in management. Diagnostics (Basel). 2020;10(11):907. doi:10.3390/diagnostics10110907 3. Bile acids. Labcorp.com. Accessed May 23, 2023. https://www.labcorp.com/tests/010330/bile-acids 4. Jahnel J, Zöhrer E, Scharnagl H, Erwa W, Fauler G, Stojakovic T. Reference ranges of serum bile acids in children and adolescents. Clin Chem Lab Med. 2015;53(11):1807-1813. doi:10.1515/cclm-2014-1273 5. LIVMARLI® (maralixibat) oral solution. Prescribing Information. 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.