KEGG   DRUG: Cipaglucosidase alfa
Entry
D11798                      Drug                                   
Name
Cipaglucosidase alfa (USAN);
Cipaglucosidase alfa (genetical recombination) (JAN);
Cipaglucosidase alfa-atga;
Pombiliti (TN)
Product
POMBILITI (AMICUS THERAPEUTICS US)
Formula
C4489H6817N1197O1298S32
Exact mass
99284.5283
Mol weight
99346.54
Sequence
QQGASRPGPR DAQAHPGRPR AVPTQCDVPP NSRFDCAPDK AITQEQCEAR GCCYIPAKQG
LQGAQMGQPW CFFPPSYPSY KLENLSSSEM GYTATLTRTT PTFFPKDILT LRLDVMMETE
NRLHFTIKDP ANRRYEVPLE TPHVHSRAPS PLYSVEFSEE PFGVIVRRQL DGRVLLNTTV
APLFFADQFL QLSTSLPSQY ITGLAEHLSP LMLSTSWTRI TLWNRDLAPT PGANLYGSHP
FYLALEDGGS AHGVFLLNSN AMDVVLQPSP ALSWRSTGGI LDVYIFLGPE PKSVVQQYLD
VVGYPFMPPY WGLGFHLCRW GYSSTAITRQ VVENMTRAHF PLDVQWNDLD YMDSRRDFTF
NKDGFRDFPA MVQELHQGGR RYMMIVDPAI SSSGPAGSYR PYDEGLRRGV FITNETGQPL
IGKVWPGSTA FPDFTNPTAL AWWEDMVAEF HDQVPFDGMW IDMNEPSNFI RGSEDGCPNN
ELENPPYVPG VVGGTLQAAT ICASSHQFLS THYNLHNLYG LTEAIASHRA LVKARGTRPF
VISRSTFAGH GRYAGHWTGD VWSSWEQLAS SVPEILQFNL LGVPLVGADV CGFLGNTSEE
LCVRWTQLGA FYPFMRNHNS LLSLPQEPYS FSEPAQQAMR KALTLRYALL PHLYTLFHQA
HVAGETVARP LFLEFPKDSS TWTVDHQLLW GEALLITPVL QAGKAEVTGY FPLGTWYDLQ
TVPVEALGSL PPPPAAPREP AIHSEGQWVT LPAPLDTINV HLRAGYIIPL QGPGLTTTES
RQQPMALAVA LTKGGEARGE LFWDDGESLE VLERGAYTQV IFLARNNTIV NELVRVTSEG
AGLQLQKVTV LGVATAPQQV LSNGVPVSNF TYSPDTKVLD ICVSLLMGEQ FLVSWC
(Disulfide bridge: 26-53, 36-52, 47-71, 477-502, 591-602)
  Type
Peptide
Remark
ATC code: A16AB23
Product: D11798<US>
Efficacy
Enzyme replacement (alpha-glucosidase)
  Disease
Pompe disease [DS:H01940]
  Type
Enzyme replacement therapy product
Comment
Treatment of Pompe disease
Target
GAA* [HSA_VAR:2548v1] [HSA:2548] [KO:K12316]
  Pathway
hsa00052  Galactose metabolism
hsa00500  Starch and sucrose metabolism
hsa04142  Lysosome
  Network
nt06017  Glycogen metabolism
Brite
Anatomical Therapeutic Chemical (ATC) classification [BR:br08303]
 A ALIMENTARY TRACT AND METABOLISM
  A16 OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
   A16A OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
    A16AB Enzymes
     A16AB23 Cipaglucosidase alfa
      D11798  Cipaglucosidase alfa (USAN) <US>
Target-based classification of drugs [BR:br08310]
 Enzymes
  Hydrolases (EC3)
   Glycosidases
    GAA* [HSA_VAR:2548v1]
     D11798  Cipaglucosidase alfa (USAN) <US>
New drug approvals in the USA [br08319.html]
 New molecular entities and new therapeutic biological products
  D11798
New drug approvals in Europe [br08329.html]
 European public assessment reports (EPAR) authorised medicine
  D11798
New drug approvals in the USA, Europe and Japan [br08328.html]
 Approval dates by FDA, EMA and PMDA
  D11798
Pharmacogenomic biomarkers [br08341.html]
 Germline mutations in genetic disorder treatments
  D11798
Other DBs
CAS: 2359727-71-0
PubChem: 405226664
LinkDB

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