WHA

Amvuttra (vutrisiran)

Indications for Prior Authorization

Amvuttra (vutrisiran)
  • For diagnosis of Hereditary transthyretin-mediated amyloidosis
    Indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

Criteria

Amvuttra

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)

  • Diagnosis of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with polyneuropathy
    • AND
  • Presence of a transthyretin (TTR) mutation (e.g., V30M) as detected by an FDA-approved test or a test performed at a facility approved by Clinical Laboratory Improvement Amendments (CLIA) [1-3]
    • AND
  • One of the following [1-4, A, B]:
    • Patient has a baseline polyneuropathy disability (PND) score less than or equal to IIIb
    • Patient has a baseline familial amyloidotic polyneuropathy (FAP) stage of 1 or 2
    • Patient has a baseline neuropathy impairment score (NIS) greater than or equal to 5 and less than or equal to 130
    • Patient has a baseline Karnofsky Performance Status score greater than or equal to 60%
    AND
  • Presence of clinical signs and symptoms of the disease (e.g., peripheral/autonomic neuropathy, walking ability, quality of life) [1-3]
    • AND
  • Patient has not had a liver transplant [2-3]
    • AND
  • Requested drug is not used in combination with a TTR silencer (e.g.,Tegsedi) or a TTR stabilizer (e.g., Vyndaqel)
    • AND
  • Prescribed by or in consultation with a neurologist
Amvuttra

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)

  • Patient demonstrates positive clinical response to therapy (e.g., improved quality of life, decreased in serum TTR level)
    • AND
  • One of the following [1-4, A, B]:
    • Patient continues to have a polyneuropathy disability (PND) score less than or equal to IIIb
    • Patient continues to have a familial amyloidotic polyneuropathy (FAP) stage of 1 or 2
    • Patient continues to have a neuropathy impairment score (NIS) greater than or equal to 5 and less than or equal to 130
    • Patient continues to have a Karnofsky Performance Status score greater than or equal to 60%
    AND
  • Requested drug is not used in combination with a TTR silencer (e.g.,Tegsedi) or a TTR stabilizer (e.g., Vyndaqel)
    • AND
  • Patient has not had a liver transplant [2-3]

  • Guideline ID
    GL-164867

  • Formulary
    premium

  • Effective date
    Mar 1, 2025

  • P&T approval date
    Sep 21, 2022

P & T Revisions

2025-02-06, 2024-09-18, 2023-09-29, 2023-08-02, 2022-08-09

  1. Amvuttra Prescribing Information. Alnylam Pharmaceuticals, Inc. Cambridge, MA. January 2023.
  2. Adams D, Tournev IL, Taylor MS, et al. Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial. Amyloid. 2022;1-9. Available at https://www.tandfonline.com/doi/full/10.1080/13506129.2022.2091985. Accessed August 4, 2022.
  3. Adams D, Suhr OB, Dyck PJ, et al. Trial design and rationale for APOLLO, a phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy. BMC Neurol. 2017;17:181.
  4. Amvuttra. European Medicines Agency Web Site. https://www.ema.europa.eu/en/medicines/human/summaries-opinion/amvuttra. Published July 21, 2022. Accessed August 4, 2022.
  5. Velez-Santamaria, V.,Nedkova-Hristova, V., et al. Hereditary Transthyretin Amyloidosis with Polyneuropathy: Monitoring and Management. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9789700/. Accessed August 2, 2023.
  6. Carroll, A., Dyck, P., et al. Novel approaches to diagnosis and management of hereditary transthyretin amyloidosis. Available at: https://jnnp.bmj.com/content/93/6/668. Accessed August 2, 2023.
  1. The efficacy of vutrisiran was demonstrated in a phase 3, open label, randomized clinical trial (HELIOS-A) in which efficacy endpoints for vutrisiran were compared to an external placebo group from the APOLLO trial. Similar recruitment criteria were used for both HELIOS-A and APOLLO which resulted in similar baseline characteristics between the treatment groups in HELIOS-A and the placebo group in APOLLO [2-3].
  2. Baseline characteristics in HELIOS-A included 70% of patients with stage 1 disease and 30% of patients with stage 2 disease [1-2]. The European Medicines Agency lists the full indication for Amvuttra as treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy [4].
  • 2025-02-06: Updated FDA/ CLIA language & positive response to therapy in line with other aTTR agents
  • 2024-09-18: 2024 annual review. Background Updates. Addition of criterion 'patient has not had a liver transplant' to the reauth for consistency throughout PA. Addition of criterion to confirm Amvuttra is not used in combination with a TTR silencer or a TTR stabilizer.
  • 2023-09-29: Program update to standard reauthorization language. No changes to clinical intent.
  • 2023-08-02: 2023 Annual Review.
  • 2022-08-09: 2022 New UM PA Criteria