PharmaKB logo
Company Reports
Company Reports
Drug Reports
Drug Reports
Disease Reports
Disease Reports
AboutPricing
Drug ReportsCipaglucosidase alfa
Pombiliti(cipaglucosidase alfa)
Pombiliti (cipaglucosidase alfa) is an enzyme pharmaceutical. Cipaglucosidase alfa was first approved as Pombiliti on 2023-03-20. It is used to treat glycogen storage disease type II in the USA. It has been approved in Europe to treat glycogen storage disease type II.
Download report
Favorite
Events Timeline
Commercial
Clinical
Drug
Target
Variants
Financial
Trends
Safety
Events Timeline
5D
1M
3M
6M
YTD
1Y
2Y
5Y
Max
Events
FDA approval date
EMA approval date
Patent expiration date
Study first post date
Last update post date
Start date
Primary completion date
Completion date
Results first post date
Plot placeholder
Mock data
Subscribe for the real data
Commercial
Therapeutic Areas
Therapeutic Area
MeSH
nervous system diseasesD009422
hereditary congenital and neonatal diseases and abnormalitiesD009358
nutritional and metabolic diseasesD009750
Trade Name
FDA
EMA
Pombiliti
Drug Products
FDA
EMA
Reference product - 351(a)
Reference product - 351(a)
Interchangeable product - 351(k)
Interchangeable product - 351(k)
Biosimilar product - 351(k)
Biosimilar product - 351(k)
Cipaglucosidase alfa
Tradename
Proper name
Company
Number
Date
Products
Pombiliticipaglucosidase alfa-atgaAmicus TherapeuticsN-761204 RX2023-09-28
1 products
Labels
FDA
EMA
Brand Name
Status
Last Update
pombilitiBiologic Licensing Application2024-07-29
Indications
FDA
EMA
Agency Specific
FDA
EMA
No data
Patent Expiration
No data
ATC Codes
A: Alimentary tract and metabolism drugs
— A16: Other alimentary tract and metabolism products in atc
— A16A: Other alimentary tract and metabolism products in atc
— A16AB: Enzymes for alimentary tract and metabolism
— A16AB23: Cipaglucosidase alfa
HCPCS
No data
Clinical
Clinical Trials
51 clinical trials
View more details
Plot placeholder
Mock data
Subscribe for the real data
Indications Phases 4
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Glycogen storage disease type iiD006009Orphanet_365E74.02108571035
Glycogen storage diseaseD006008—E74.0—126211
Type 2 diabetes mellitusD003924EFO_0001360E11——2518
Diabetes mellitusD003920HP_0000819E08-E13——25—7
Glycogen storage disease type iiiD006010Orphanet_366E74.03——13—4
Liver diseasesD008107HP_0002910K70-K77———1—1
Non-alcoholic fatty liver diseaseD065626EFO_0003095K75.81———1—1
Fatty liverD005234EFO_0003934————1—1
Type 1 diabetes mellitusD003922EFO_0001359E10———1—1
Indications Phases 3
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
DiseaseD004194EFO_0000408R69——1——1
Deficiency diseasesD003677EFO_1001067E63——1——1
Indications Phases 2
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
HypotensionD007022EFO_0005251I95—1———1
SyncopeD013575HP_0012668G90.01—1———1
Amyotrophic lateral sclerosisD000690HP_0007354G12.21—1———1
Motor neuron diseaseD016472EFO_0003782G12.2—1———1
SclerosisD012598———1———1
Indications Phases 1
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
HypersensitivityD006967HP_0012393T78.401————1
Healthy volunteers/patients———1————1
Indications Without Phase
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
AtaxiaD001259—R27.0————22
Cerebellar ataxiaD002524HP_0001251—————22
OverweightD050177—E66.3————11
Deglutition disordersD003680HP_0002015R13.1————11
Metabolic diseasesD008659EFO_0000589E88.9————11
Lysosomal storage diseasesD016464——————11
SarcopeniaD055948EFO_1000653M62.84————11
Duchenne muscular dystrophyD020388EFO_0000429—————11
Muscular dystrophiesD009136HP_0003560G71.0————11
MyalgiaD063806HP_0003326M79.1————11
Show 4 more
Epidemiology
Epidemiological information for investigational and approved indications
View more details
Drug
General
Drug common nameCipaglucosidase alfa
INNcipaglucosidase alfa
Description
Cipaglucosidase alfa, sold under the brand name Pombiliti, and used in combination with miglustat, is a medication used for the treatment of glycogen storage disease type II (Pompe disease). Cipaglucosidase alfa is a recombinant human acid α-glucosidase enzyme replacement therapy that provides an exogenous source of acid α-glucosidase.
Classification
Enzyme
Drug classenzymes
Image (chem structure or protein)Loading
Structure (InChI/SMILES or Protein Sequence)—
Identifiers
PDB—
CAS-ID—
RxCUI—
ChEMBL IDCHEMBL4594329
ChEBI ID—
PubChem CID—
DrugBankDB16708
UNII ID4SED7F4BSG (ChemIDplus, GSRS)
Target
No data
Variants
No data
Financial
No data
Trends
PubMed Central
Top Terms for Disease or Syndrome:
Plot placeholder
Mock data
Subscribe for the real data
Additional graphs summarizing 76 documents
View more details
Safety
Black-box Warning
Black-box warning for: Pombiliti
Adverse Events
Top Adverse Reactions
Plot placeholder
Mock data
Subscribe for the real data
4 adverse events reported
View more details
© 2020-2024 Collaborative Drug Discovery Inc. (CDD) | Terms of Use