WHA

Bylvay (odevixibat)

Indications for Prior Authorization

Bylvay (odevixibat)
  • For diagnosis of Pruritus associated with progressive familial intrahepatic cholestasis (PFIC)
    Indicated for the treatment of pruritus in patients 3 months of age and older with progressive familial intrahepatic cholestasis (PFIC).

    Limitation of Use: Bylvay may not be effective in PFIC type 2 patients with ABCB11 variants resulting in non-functional or complete absence of bile salt export pump protein (BSEP-3).

  • For diagnosis of Alagille syndrome
    Indicated for the treatment of cholestatic pruritus in patients 12 months of age and older with Alagille syndrome.

Criteria

Bylvay

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)
For diagnosis of Progressive Familial Intrahepatic Cholestasis (PFIC)

  • Diagnosis of progressive familial intrahepatic cholestasis (PFIC) type 1, 2, or 3 confirmed by one of the following: [B-D, 2]
    • Diagnostic test (e.g., liver function test, liver ultrasound and biopsy, bile analysis)
    • Genetic Testing
    AND
  • Patient is experiencing both of the following: [1]
    • Moderate to severe pruritus
    • Patient has a serum bile acid concentration above the upper limit of the normal reference for the reporting laboratory
    AND
  • Patient is 3 months of age or older
    • AND
  • Patient has had an inadequate response to at least two of the following treatments used for the relief of pruritus: [6]
    • Ursodeoxycholic acid (e.g., Ursodiol)
    • Antihistamines (e.g., diphenhydramine, hydroxyzine)
    • Rifampin
    • Bile acid sequestrants (e.g., Questran, Colestid, Welchol)
    AND
  • Prescribed by or in consultation with a hepatologist or gastroenterologist
Bylvay

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)
For diagnosis of Progressive Familial Intrahepatic Cholestasis (PFIC)

  • Patient demonstrates positive clinical response to therapy (e.g., reduced serum bile acids, improved pruritus)
Bylvay

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)
For diagnosis of Alagille Syndrome (ALGS)

  • Both of the following:
    • Diagnosis of Alagille Syndrome (ALGS)
      • AND
    • Molecular genetic testing confirms mutations in the JAG1 or NOTCH2 gene [E, 7, 9]
    AND
  • Patient is experiencing both of the following: [10]
    • Moderate to severe cholestatic pruritus
    • Patient has a serum bile acid concentration above the upper limit of the normal reference for the reporting laboratory
    AND
  • Patient has had an inadequate response to at least two of the following treatments used for the relief of pruritus: [F, 7-8]
    • Ursodeoxycholic acid (e.g., Ursodiol)
    • Antihistamines (e.g., diphenhydramine, hydroxyzine)
    • Rifampin
    • Bile acid sequestrants (e.g., Questran, Colestid, Welchol)
    AND
  • Patient is 12 months of age or older
    • AND
  • Prescribed by or in consultation with a hepatologist or gastroenterologist
Bylvay

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)
For diagnosis of Alagille Syndrome (ALGS)

  • Patient demonstrates positive clinical response to therapy (e.g., reduced bile acids, reduced pruritus severity score)

  • Guideline ID
    GL-220202

  • Formulary
    EHB

  • Effective date
    Mar 15, 2025

  • P&T approval date
    Sep 15, 2021

P & T Revisions

2025-03-13, 2024-08-14, 2023-08-22, 2023-07-21, 2022-09-09, 2022-03-28, 2021-11-19, 2021-08-23, 2021-08-18

  1. Bylvay (odevixibat) [prescribing information]. Albireo Pharma Inc. Boston, MA. June 2023.
  2. PFIC Advocacy and Resource Network, Inc. Available at https://www.pfic.org/types-and-subtypes-of-pfic/ Accessed August 5, 2021.
  3. Bylvay (odevixibat) [prescribing information]. Available at https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/215498s000lbl.pdf?utm_medium=email&utm_source=govdelivery. Accessed August 5, 2021.
  4. Lexicomp [database online]. Available at www.uptodate.com/contents/odevixibat-drug-information?search=bylvay&source=panel_search_result&selectedTitle=1~1&usage_type=panel&kp_tab=drug_general&display_rank=1. Last accessed August 5, 2021.
  5. www.albireopharma.com/patients-families/progressive-familial-intrahepatic-cholestasis-pfic. Last accessed August 12, 2021.
  6. Clinical Consult with Sirish Palle M.D. October 29, 2021.
  7. 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
  8. 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.
  9. 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/
  10. ClinicalTrials.gov: Available at: https://www.clinicaltrials.gov/study/NCT04674761?term=NCT04674761&rank=1. Accessed July 12, 2023.
  11. 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
  1. If there is no improvement in pruritus after 3 months, the dosage may be increased in 40 mcg/kg increments up to 120 mcg/kg once daily not to exceed a total daily dose of 6 mg [3].
  2. The efficacy of BYLVAY was evaluated in Trial 1 (NCT03566238), a 24-week, randomized, double-blind, placebo-controlled trial. Trial 1 was conducted in 62 pediatric patients, aged 6 months to 17 years, with a confirmed molecular diagnosis of PFIC type 1 or type 2, and presence of pruritus at baseline. [3]
  3. Trial 2 is a 72-week, open-label, single-arm trial in PFIC type 1, 2, and 3 patients. [3]
  4. Diagnostic testing may include liver functions tests, liver ultrasound and biopsy, and/or bile analysis. Genetic testing may be used in selected patients to confirm diagnosis and distinguish type. All 3 subtypes of PFIC have increased serum bile acid levels. [5]
  5. Alagille Syndrome is an autosomal dominant disease with variable expressivity, caused by heterozygous mutations in either JAG1 or NOTCH2. The vast majority of cases are due to JAG1 mutations accounting for 94%, and NOTCH2 mutations in additional 2–4%. [7]
  6. The management of pruritus in ALGS is challenging, and a variety of therapies are often used. These include antihistamines, rifampin, ursodeoxycholic acid, cholestyramine, naltrexone, and sertraline. Clinical experience suggests that these drugs have variable efficacy in reducing pruritus; however, no prospective clinical trials has quantified the effect of any of these therapies, either alone or in combination. [8]
  • 2025-03-13: Addition of EHB formulary to guideline.
  • 2024-08-14: 2024 Annual Review.
  • 2023-08-22: Program update to standard reauthorization language. No changes to clinical intent.
  • 2023-07-21: 2023 Annual Review (PFIC) & New Indication for Alagille syndrome.
  • 2022-09-09: 2022 Annual Review
  • 2022-03-28: Update Guideline
  • 2021-11-19: 2022 PA UM Review
  • 2021-08-23: New PA Um Criteria
  • 2021-08-18: Bylvay New PA Criteria