Wainua (eplontersen subcutaneous injection) — Medical Mutual

Initial criteria

• Patient is age ≥ 18 years; AND
• Patient has a transthyretin mutation as confirmed by genetic testing [documentation required]; AND
• Presence of polyneuropathy characterized by ONE of the following (i, ii, or iii): i. Baseline Neuropathy Impairment Scale+7 (mNIS+7) OR ii. Baseline Norfolk Quality of Life Diabetic Neuropathy (QoL-DN) Questionnaire OR iii. Baseline FAP Stage 1 or 2; AND
• Patient has polyneuropathy as demonstrated by at least TWO of the following criteria (i, ii, or iii): i. Subjective patient symptoms are suggestive of neuropathy ii. Abnormal nerve conduction studies are consistent with polyneuropathy iii. Abnormal neurological examination is suggestive of neuropathy; AND
• Patient’s peripheral neuropathy is attributed to hATTR/FAP and other causes of neuropathy have been excluded; AND
• The requested medication will not be used concurrently with Onpattro (patisiran) injections, Amvuttra (vutrisiran) injections, or tafamadis products (Vynaqel and Vyndamax); AND
• Patient does not have a history of liver transplantation; AND
• The medication is prescribed by or in consultation with a neurologist, geneticist, or a physician who specializes in the treatment of amyloidosis.

Reauthorization criteria

• Patient is age ≥ 18 years; AND
• Patient has experienced a positive clinical response to Wainua (e.g., improved neurologic impairment, motor function, cardiac function, quality of life assessment, serum TTR levels, etc.); AND
• Improvement from baseline or stabilization of ONE of the following (i, ii, or iii): i. Baseline Neuropathy Impairment Scale+7 (mNIS+7) ii. Baseline Norfolk Quality of Life Diabetic Neuropathy (QoL-DN) Questionnaire iii. Baseline FAP Stage 1 or 2; AND
• The requested medication will not be used concurrently with Onpattro (patisiran) injections, Amvuttra (vutrisiran) injections, Tegsedi (inotersen) injections, or tafamadis products (Vynaqel and Vyndamax); AND
• Absence of unacceptable toxicity from the drug (examples include ocular symptoms related to hypovitaminosis A); AND
• The medication is prescribed by or in consultation with a neurologist, geneticist, or a physician who specializes in the treatment of amyloidosis.

Approval duration

initial: 6 months; reauth: 1 year