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ONPATTRO® (patisiran)—the first and only FDA-approved RNAi therapeutic for the polyneuropathy of hATTR amyloidosis1

What is ONPATTRO?

ONPATTRO is a double-stranded small interfering ribonucleic acid (siRNA) formulated for targeted delivery to hepatocytes, the primary source of transthyretin (TTR) protein in circulation1,2

TTR protein in a patient with hATTR amyloidosis2

Mechanism of action for ONPATTRO® (patisiran)

ONPATTRO targets the liver

Mechanism of action for ONPATTRO® (patisiran)

mRNA=messenger ribonucleic acid; RNAi=ribonucleic acid interference.

Reducing the pathogenic TTR protein leads to a reduction in amyloid deposits in tissues.1,3,4

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References:

  1. ONPATTRO [package insert]. Cambridge, MA: Alnylam Pharmaceuticals, Inc; 2018.
  2. Gertz MA. Am J Manag Care. 2017;23(7 suppl):S107-S112.
  3. Adams D, Coelho T, Conceicao I, et al. Slides presented at: American Academy of Neurology; April 26, 2017; Boston, MA.
  4. Tsuchiya A, Yazaki M, Kametani F, et al. Liver Transpl. 2008;14(4):563-570.