Patisiran (Onpattro™)

Onpattro (patisiran) is covered only for the FDA‑approved indication—treatment of polyneuropathy due to hereditary transthyretin‑mediated (hATTR) amyloidosis in adults—and non‑FDA uses and patients with prior liver transplantation are excluded. Approval (up to 12 months) requires genetic confirmation of hATTR, documented symptomatic polyneuropathy (e.g., clinical exam/EMG/NCV), age ≥18, prescription by or consultation with an amyloidosis specialist (neurologist/geneticist), and dosing per label 0.3 mg/kg IV every 3 weeks (max 30 mg).

"Onpattro is indicated for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults."

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