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.
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Commercial
Therapeutic Areas
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 |
|---|---|---|---|---|---|
| Pombiliti | cipaglucosidase alfa-atga | Amicus Therapeutics | N-761204 RX | 2023-09-28 | 1 products |
Labels
FDA
EMA
Brand Name | Status | Last Update |
|---|---|---|
| pombiliti | Biologic Licensing Application | 2024-11-27 |
Indications
FDA
EMA
Indication | Ontology | MeSH | ICD-10 |
|---|---|---|---|
| glycogen storage disease type ii | Orphanet_365 | D006009 | E74.02 |
Agency Specific
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Clinical
Clinical Trials
51 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 ii | D006009 | Orphanet_365 | E74.02 | 2 | 2 | 4 | — | 4 | 11 |
| Glycogen storage disease | D006008 | — | E74.0 | — | — | 1 | — | 1 | 2 |
Indications Phases 2
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Indications Phases 1
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Hypersensitivity | D006967 | EFO_0003785 | T78.40 | 1 | — | — | — | — | 1 |
Indications Without Phase
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Metabolic diseases | D008659 | EFO_0000589 | E88.9 | — | — | — | — | 1 | 1 |
| Lysosomal storage diseases | D016464 | — | — | — | — | — | — | 1 | 1 |
Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
| Drug common name | Cipaglucosidase alfa |
| INN | cipaglucosidase 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 class | enzymes |
| Image (chem structure or protein) |  |
| Structure (InChI/SMILES or Protein Sequence) | — |
Identifiers
| PDB | — |
| CAS-ID | — |
| RxCUI | — |
| ChEMBL ID | CHEMBL4594329 |
| ChEBI ID | — |
| PubChem CID | — |
| DrugBank | DB16708 |
| UNII ID | 4SED7F4BSG (ChemIDplus, GSRS) |
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Additional graphs summarizing 97 documents
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Safety
Black-box Warning
Black-box warning for: Pombiliti
Adverse Events
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32 adverse events reported
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