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DrugOnpattro® (patisiran injection, lipid comples) [Alnylam Pharmaceuticals, Inc.]

April 2020

Therapeutic area - Amyloidosis Agents

Initial approval criteria

  • Patient must be ≥ 18 years of age AND
  • Patient has a definitive diagnosis of hATTR amyloidosis/FAP as documented by amyloid deposition on tissue biopsy and identification of a pathogenic TTR variant using molecular genetic testing AND
  • Onpattro is used for the treatment of polyneuropathy as demonstrated by at least 2 of the following criteria:
    • Subjective patient symptoms are suggestive of neuropathy
    • Abnormal nerve conduction studies are consistent with polyneuropathy
    • Abnormal neurological examination is suggestive of neuropathy
    • Patient’s peripheral neuropathy is attributed to hATTR/FAP and other causes of neuropathy have been ruled out
    • Baseline in strength/weakness has been documented using an objective clinical measuring tool (e.g., Medical Research Council [MRC] muscle strength, etc.)
    • Patient has not been the recipient of an orthotopic liver transplant (OLT)
    • Patient is receiving vitamin A supplementation at the recommended daily allowance (e.g., 400-900 retinol activity equivalents [RAE])
  • Initial approval is for 6 months

Renewal criteria

  • Patient continues to meet initial criteria AND
  • Absence of unacceptable toxicity from the drug (including severe infusion-related reactions, ocular symptoms related to hypovitaminosis A, etc.) AND
  • Renewal requests for Onpattro coverage at month 18 and beyond, disease response compared to pre-treatment baseline as evidenced by stabilization or improvement in 1 or more of the following:
    • Signs and symptoms of neuropathy OR
    • MRC muscle strength
  • Renewal approval is for 6 months

Quantity limits

  • 30 mg (3 vials) every 3 weeks


MHCP Provider Call Center 651-431-2700 or 800-366-5411

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