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Tabel 1 Basic information and pharmakokinetics

From: Are we creating a new phenotype? Physiological barriers and ethical considerations in the treatment of hereditary transthyretin-amyloidosis

  Tafamidis (Vyndaqel™/Vyndamax™) Patisiran (Onpattro™) Vutrisirana Inotersen (Tegsedi™) Eplontersena TTR
Dosage 20/61 mg 0.3 mg per kg body weight 25 mg 300 mg 45 mg NA
Application oral i.v. s.c. s.c. s.c. NA
Frequency 1×/d 1×/3 weeks 1×/3 months 1×/week 1×/4 weeks NA
Effect site Liver/blood Liver Liver Liver Liver NA
Uptake mechanism/receptors Unspecific Unspecific Specific trough GalNaC modification via ASGPR Unspecific Specific trough GalNaC modification via ASGPR NA
Molecule size [D] 308.12 14,304 MD 7600.8 9046.1 55 K, 16 K g/mol
Plasma t1/2 24 h 0.79 h 4.8 h 3.9 h 1.6 h 48 h
Tissue distribution Liver and plasma Liver  lymphatic tissues > kidneys, lungs, heart, adrenals Liver, kidneys, injection site Liver, kidney, lymphatic tissues, injection site, bone marrow Liver, kidney, injection site Plasma, CSF, eyes, liver, kidney, pancreas
BBB passage Partial (1.5%) None None None None 2–10% synthesis in choroid plexus and retina
  1. Synoptic summary on the three approved drugs tafamidis, patisiran, and inotersen, and the two drugs vutrisiran and eplontersen that are currently in phase III trials. Data were provided by the respective pharma companies (Pfizer, Alnylam, and Ionis). If the exact information was not being publicly available at the time when this paper was written (09/2021) or provided upon request, we indicated this with MD (missing data). For comparison, we provide similar information on the TTR protein on the right. ASGPR, asialoglycoprotein receptor receptor; BBB, blood–brain barrier; CSF, cerebrospinal fluid; GalNac, NA, N-Acetylgalactosamine; h, hour(s); i.v., intraveneous; NA, not applicable; s.c., subcutaneous; t1/2, half-life
  2. aDrug not (yet) approved in Europe or in the United States of America