Pharmacy Prior Authorization Criteria

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.

• For diagnosis of Transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM)
  Indicated for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits.

Criteria

Amvuttra

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)
For diagnosis of Hereditary transthyretin-mediated amyloidosis with polyneuropathy (hATTR-PN)

• 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)
For diagnosis of Hereditary transthyretin-mediated amyloidosis with polyneuropathy (hATTR-PN)

• 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]

Amvuttra

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)
For diagnosis of Transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM)

• Diagnosis of transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM)
AND
• One of the following:
• Presence of a transthyretin (TTR) mutation (e.g., V122I) as detected by an FDA-approved test or a test performed at a facility approved by Clinical Laboratory Improvement Amendments (CLIA)
OR
• Cardiac or noncardiac tissue biopsy demonstrating histologic confirmation of TTR amyloid deposits
OR
• Both of the following:
• Cardiac magnetic resonance imaging or scintigraphy scan suggestive of amyloidosis
• Absence of light-chain amyloidosis
AND
• Patient has New York Heart Association (NYHA) Functional Class I, II, or III heart failure
AND
• Requested drug is not used in combination with a TTR silencer (e.g.,Onpattro)
AND
• Prescribed by or in consultation with a cardiologist

Amvuttra

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)
For diagnosis of Transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM)

• Patient continues to have New York Heart Association (NYHA) Functional Class I, II, or III heart failure
AND
• Requested drug is not used in combination with a TTR silencer (e.g.,Onpattro)
AND
• Prescribed by or in consultation with a cardiologist

P & T Revisions

2025-04-04, 2025-02-06, 2024-09-18, 2023-09-29, 2023-08-02, 2022-08-09

References

1. Amvuttra Prescribing Information. Alnylam Pharmaceuticals, Inc. Cambridge, MA. March 2025.
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.
7. Fontana M, Berk JL, Gillmore JD, et al. Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy. N Engl J Med. 2025;392(1):33-44. doi:10.1056/NEJMoa2409134

---

End Notes

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].

---

Revision History

• 2025-04-04: Added criteria for new indication of Transthyretin-mediated amyloidosis with cardiomyopathy (ATTR-CM). Background updates.
• 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

---