WAINUA (eplontersen)

WAINUA (eplontersen)

Self-Administration – subcutaneous injection

Diagnosis considered for coverage:

Hereditary transthyretin-mediated (hATTR) amyloidosis: Indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
 

Coverage Criteria:

For diagnosis of hATTR amyloidosis:

• Diagnosis of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with polyneuropathy; AND 
• Prescribed by or in consultation with a neurologist; AND
• Patient has a transthyretin (TTR) mutation (e.g., V30M); AND 
• ONE of the following: 
  • 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); AND 
• Patient has not had a liver transplant

Reauthorization Criteria:

For diagnosis of hATTR amyloidosis:

• Patient demonstrates positive clinical response to therapy as evidenced by an improvement in clinical signs and symptoms from baseline (e.g., neuropathy, quality of life, lower serum TTR level); AND
• One of the following: 
  • 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 

Coverage Duration:

• Initial: 1 year
• Reauthorization: 1 year

Dosing:

For diagnosis of hATTR amyloidosis:

• The recommended dosage is 45 mg administered by subcutaneous injection once monthly.
• Supplementation at the recommended daily allowance of vitamin A is advised because treatment leads to a decrease in serum vitamin A levels.
   

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: 

• Hereditary transthyretin amyloidosis (ATTRv) is a progressive debilitating, fatal genetic disorder that presents with neuropathy and/or cardiomyopathy.
• Guidelines recommend treatment based on presence of neuropathy or cardiomyopathy. 

Policy Updates:

09/01/2024 – New policy for Wainua approved by WHA P&T Committee. (P&T, 08/20/2024)
 

References:

1. Wainua Prescribing Information. AstraZeneca Pharmaceuticals LP, Wilmington, DE 19850. December 2023 
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.