Pharmacy Prior Authorization Criteria

Wainua (eplontersen)

Indications for Prior Authorization

Wainua (eplontersen)

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

Criteria

Wainua

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]
AND
• One of the following [2, 3]:
• 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 10 and less than or equal to 130
• Patient has a baseline Karnofsky Performance Status score greater than 50%
AND
• Presence of clinical signs and symptoms of the disease (e.g., neuropathy, quality of life) [2]
AND
• Patient has not had a liver transplant [2, 3]
AND
• Requested drug is not used in combination with a TTR silencer (e.g., Amvuttra) or a TTR stabilizer (e.g., Vyndaqel)
AND
• Prescribed by or in consultation with a neurologist

Wainua

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)

• Patient demonstrates positive clinical response to therapy (e.g., improved quality of life, decreased serum TTR level)
AND
• One of the following [2, 3]:
• 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 10 and less than or equal to 130
• Patient continues to have a Karnofsky Performance Status score greater than 50%
AND
• Patient has not had a liver transplant [2, 3]
AND
• Requested drug is not used in combination with a TTR silencer (e.g., Amvuttra) or a TTR stabilizer (e.g., Vyndaqel)

P & T Revisions

2025-04-02, 2025-02-06, 2024-09-04, 2024-03-06

References

1. Wainua Prescribing Information. AstraZeneca Pharmaceuticals LP, Wilmington, DE 19850. September 2024.
2. Coelho T, Ando Y, Benson MD et al. Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-LRx (ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy. Neurol Ther. 2021 Jun;10(1):375-389. doi: 10.1007/s40120-021-00235-6. Epub 2021 Feb 26
3. Study Details | NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy | ClinicalTrials.gov https://clinicaltrials.gov/study/NCT04136184. Accessed February 9, 2024.

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Revision History

• 2025-04-02: 2025 Annual Review. No criteria changes. Updated references.
• 2025-02-06: Updated to standard ORx language. No change to clinical intent.
• 2024-09-04: Updated guideline.
• 2024-03-06: New Program for Wainua

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