Onpattro (patisiran) — Medical Mutual

Initial criteria

• Patient is age ≥ 18 years; AND
• Patient is receiving supplementation with vitamin A at the recommended daily allowance; AND
• Must not be used in combination with other transthyretin (TTR) reducing or stabilizing agents (e.g., inotersen, tafamidis, vutrisiran, eplontersen, etc.); AND
• Patient has a definitive diagnosis of hATTR amyloidosis as documented in a proband with suggestive findings (including imaging or histopathology findings of amyloidosis) and a heterozygous pathogenic (or likely pathogenic) variant in TTR identified by molecular genetic testing; AND
• Used for the treatment of polyneuropathy as demonstrated by at least TWO of the following criteria: subjective patient symptoms are suggestive of neuropathy OR abnormal nerve conduction studies are consistent with polyneuropathy OR abnormal neurological examination is suggestive of neuropathy; AND
• Patient’s peripheral neuropathy is attributed to hATTR and other causes of neuropathy have been excluded; AND
• Baseline in strength/weakness has been documented using an objective clinical measuring tool (e.g., Medical Research Council (MRC) muscle strength, etc.); AND
• Patient has not been the recipient of an orthotopic liver transplant (OLT)

Reauthorization criteria

• Patient continues to meet the universal and other indication-specific relevant criteria identified in section III; AND
• Absence of unacceptable toxicity from the drug (examples include severe infusion-related reactions, ocular symptoms related to vitamin A deficiency such as night blindness); AND
• Disease response compared to pre-treatment baseline as evidenced by stabilization or improvement in one or more of the following: signs and symptoms of neuropathy OR MRC muscle strength

Approval duration

Initial: 6 months; Renewal: 12 months