KEGG   DRUG: Eplontersen sodium
Entry
D12754                      Drug                                   
Name
Eplontersen sodium (JAN/USAN);
Wainua (TN)
Product
Formula
C296H417N77O156P20S13. 20Na
Exact mass
9040.6140
Mol weight
9046.05
Sequence
DNA, d([2′-O-(2-methoxyethyl) ]m5rU-sp-[2′-O-(2-methoxyethyl)
]m5rC-[2′-O-(2-methoxyethyl) ]m5rU-[2′-O-(2-methoxyethyl)
]m5rU-[2′-O-(2-methoxyethyl)
]rG-G-sp-T-sp-T-sp-A-sp-m5C-sp-A-sp-T-sp-G-sp-A-sp-A-sp-[2′-O-(2-methoxyethyl)
]rA-[2′-O-(2-methoxyethyl) ]m5rU-[2′-O-(2-methoxyethyl)
]m5rC-sp-[2′-O-(2-methoxyethyl) ]m5rC-sp-[2′-O-(2-methoxyethyl)
]m5rC), 5′-[26-[[2-(acetylamino)
-2-deoxy-β-D-galactopyranosyl]oxy]-14,14-bis[[3-[[6-[[2-(acetylamino)
-2-deoxy-β-D-galactopyranosyl]oxy]hexyl]amino]-3-oxopropoxy]methyl]-8,12,19-trioxo-16-oxa-7,13,20-triazahexacos-1-yl
hydrogen phosphate]
  Type
Nucleotide
Remark
ATC code: N07XX21
Product: D12754<US>
Efficacy
Amyloid fibril deposition inhibitor
  Disease
Polyneuropathy of hereditary transthyretin-mediated amyloidosis [DS:H00845]
  Type
Antisense oligonucleotide
Comment
Treatment of transthyretin-mediated amyloidosis
Target
TTR [HSA:7276] [KO:K20731] (mRNA)
  Pathway
hsa04918  Thyroid hormone synthesis
Brite
Anatomical Therapeutic Chemical (ATC) classification [BR:br08303]
 N NERVOUS SYSTEM
  N07 OTHER NERVOUS SYSTEM DRUGS
   N07X OTHER NERVOUS SYSTEM DRUGS
    N07XX Other nervous system drugs
     N07XX21 Eplontersen
      D12754  Eplontersen sodium (JAN/USAN) <US>
USP drug classification [BR:br08302]
 Genetic, Enzyme, or Protein Disorder: Replacement, Modifiers, Treatment
  Eplontersen
   D12754  Eplontersen sodium (JAN/USAN)
Target-based classification of drugs [BR:br08310]
 Transporters
  Other transporters
   Others
    TTR
     D12754  Eplontersen sodium (JAN/USAN) <US>
New drug approvals in the USA [br08319.html]
 New molecular entities and new therapeutic biological products
  D12754
New drug approvals in Europe [br08329.html]
 European public assessment reports (EPAR) authorised medicine
  D12754
New drug approvals in the USA, Europe and Japan [br08328.html]
 Approval dates by FDA, EMA and PMDA
  D12754
Other DBs
CAS: 2131025-75-5
PubChem: 497621167
LinkDB

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