LIVMARLI (maralixibat solution)

LIVMARLI (maralixibat solution) 

Self-Administration – oral

Diagnosis considered for coverage:

• Indicated for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 1 year of age and older

Coverage Criteria:

For treatment of cholestatic pruritus in ALGS:

• Dose does not exceed 380 mcg/kg once daily OR 28.5 mg (3 mL) per day; AND
• Patient is 1 year of age or older; AND
• Prescribed by or in consultation with a hepatologist; AND
• Medical records confirm a diagnosis of Alagille Syndrome (ALGS); AND
• Molecular genetic testing confirms mutations in the JAG1 or NOTCH2 gene; AND
• Documentation of member’s current weight; AND
• Documentation of ONE of the following:
  • Total serum bile acid > 3x the upper limit of normal (ULN) 
  • Conjugated bilirubin > 1 mg/dL 
  • Fat soluble vitamin deficiency otherwise unexplainable 
  • Gammaglutamyl transpeptidase (GGT) > 3x ULN; AND
• Patient is experiencing moderate to severe cholestatic pruritus; AND
• Patient has had an inadequate response to at least two of the following treatments used for the relief of pruritus:
  • Ursodeoxycholic acid (e.g., ursodiol) 
  • Antihistamines (e.g., diphenhydramine, hydroxyzine) 
  • Rifampin 
  • Bile acid sequestrants (e.g., Questran®, Colestid®, Welchol™) 

Reauthorization Criteria:

For treatment of cholestatic pruritus in ALGS:

• Dose does not exceed 380 mcg/kg once daily OR 28.5 mg (3 mL) per day; AND
• Documentation of positive clinical response to therapy (e.g., reduced bile acids, reduced pruritus severity score)

Coverage Duration: 

• Initial: 1 year
• Reauthorization: 1 year

Authorization is not covered for the following:

The use of this drug for indications not listed in this policy does not meet the coverage criteria established by the Western Health Advantage (WHA) Pharmacy and Therapeutics (P&T) Committee.

Additional Information: 

• Starting dose: 190 mcg/kg once daily
• Recommended dose: 380 mcg/kg once daily
• Maximum dose for patients above 70kg: 3 mL or 28.5 mg per day
• For patients taking bile acid binding resins, take Livmarli at least 4 hours before or 4 hours after taking a bile acid binding resin
• Warnings for liver test abnormalities, gastrointestinal adverse reactions, fat-soluble vitamin (FSV) deficiency
• The efficacy and safety in ALGS patients with clinically significant portal hypertension and in patients with decompensated cirrhosis have not been established

Policy Updates:

• 2/15/2022 – New policy approved by P&T

References:

• Livmarli Prescribing Information. Mirum Pharmaceuticals, Inc. Foster City, CA. September 2021. 
• Ayoub MD, Kamath BM. Alagille Syndrome: Diagnostic Challenges and Advances in Management. Diagnostics (Basel). 2020;10(11):907. Published 2020 Nov 6. doi:10.3390/diagnostics10110907 
• Saleh M, Kamath BM, Chitayat D. Alagille syndrome: clinical perspectives. Appl Clin Genet. 2016;9:75-82. Published 2016 Jun 30. doi:10.2147/TACG.S86420 
• ClinicalTrials.gov: https://clinicaltrials.gov/ct2/show/study/NCT02160782 Accessed October 18, 2021. 
• Emerick KM, Elias MS, Melin-Aldana H, et al. Bile composition in Alagille Syndrome and PFIC patients having Partial External Biliary Diversion. BMC Gastroenterol. 2008;8:47. Published 2008 Oct 20. doi:10.1186/1471-230X-8-47 
• Diaz-Frias J, Kondamudi NP. Alagille Syndrome. [Updated 2021 Jun 26]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2021 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK507827/ 
• Shneider BL, Spino C, Kamath BM, et al. Placebo-Controlled Randomized Trial of an Intestinal Bile Salt Transport Inhibitor for Pruritus in Alagille Syndrome. Hepatol Commun. 2018;2(10):1184-1198.