ICER reviewed new therapies for the treatment of hereditary transthyretin-related (hATTR) amyloidosis, a rare genetic condition characterized by nerve, heart, and eye damage that is currently treated through supportive measures alone. Therapies included inotersen (Akcea Therapeutics ), an antisense oligonucleotide that interferes with transthyretin production in the liver, and patisiran (OnpattroTM, Alnylam Pharmaceuticals), which inhibits transthyretin through an RNA interference pathway.

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Interventions of Interest

  • inotersen (Akcea Therapeutics )
  • patisiran (OnpattroTM, Alnylam Pharmaceuticals)

Final Documents

Below you will find the final documents from the assessment review process: