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Cipaglucosidase alfa
Pombiliti (cipaglucosidase alfa) is an enzyme pharmaceutical. Cipaglucosidase alfa was first approved as Pombiliti on 2023-03-20. It has been approved in Europe to treat glycogen storage disease type II.
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Commercial
Therapeutic Areas
Therapeutic Area
MeSH
nervous system diseasesD009422
hereditary congenital and neonatal diseases and abnormalitiesD009358
nutritional and metabolic diseasesD009750
Trade Name
FDA
EMA
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Drug Products
FDA
EMA
New Drug Application (NDA)
New Drug Application (NDA)
Abbreviated New Drug Application (ANDA)
Abbreviated New Drug Application (ANDA)
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Labels
FDA
EMA
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Indications
FDA
EMA
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Agency Specific
FDA
EMA
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Patent Expiration
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ATC Codes
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HCPCS
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Clinical
Clinical Trials
7 clinical trials
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Indications Phases 4
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Indications Phases 3
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Glycogen storage disease type iiD006009Orphanet_365E74.02114—27
Indications Phases 2
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Indications Phases 1
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Indications Without Phase
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Epidemiology
Epidemiological information for investigational and approved indications
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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)
Structure (InChI/SMILES or Protein Sequence)—
Identifiers
PDB—
CAS-ID—
RxCUI—
ChEMBL IDCHEMBL4594329
ChEBI ID—
PubChem CID—
DrugBankDB16708
UNII ID4SED7F4BSG (ChemIDplus, GSRS)
Target
Agency Approved
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Alternate
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Variants
Clinical Variant
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Financial
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Trends
PubMed Central
Top Terms for Disease or Syndrome:
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Additional graphs summarizing 11 documents
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Safety
Black-box Warning
No Black-box warning
Adverse Events
Top Adverse Reactions
0 adverse events reported
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