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 |
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Clinical
Clinical Trials
51 clinical trials
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Indications Phases 4
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 | 10 | 8 | 5 | 7 | 10 | 35 |
| Glycogen storage disease | D006008 | — | E74.0 | — | 1 | 2 | 6 | 2 | 11 |
| Type 2 diabetes mellitus | D003924 | EFO_0001360 | E11 | — | — | 2 | 5 | 1 | 8 |
| Diabetes mellitus | D003920 | EFO_0000400 | E08-E13 | — | — | 2 | 5 | — | 7 |
| Glycogen storage disease type iii | D006010 | Orphanet_366 | E74.03 | — | — | 1 | 3 | — | 4 |
| Liver diseases | D008107 | EFO_0001421 | K70-K77 | — | — | — | 1 | — | 1 |
| Non-alcoholic fatty liver disease | D065626 | EFO_0003095 | K75.81 | — | — | — | 1 | — | 1 |
| Fatty liver | D005234 | EFO_0003934 | — | — | — | — | 1 | — | 1 |
| Type 1 diabetes mellitus | D003922 | EFO_0001359 | E10 | — | — | — | 1 | — | 1 |
Indications Phases 3
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Disease | D004194 | EFO_0000408 | R69 | — | — | 1 | — | — | 1 |
| Deficiency diseases | D003677 | EFO_1001067 | E63 | — | — | 1 | — | — | 1 |
Indications Phases 2
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Hypotension | D007022 | EFO_0005251 | I95 | — | 1 | — | — | — | 1 |
| Syncope | D013575 | — | G90.01 | — | 1 | — | — | — | 1 |
| Amyotrophic lateral sclerosis | D000690 | EFO_0000253 | G12.21 | — | 1 | — | — | — | 1 |
| Motor neuron disease | D016472 | EFO_0003782 | G12.2 | — | 1 | — | — | — | 1 |
| Sclerosis | D012598 | — | — | — | 1 | — | — | — | 1 |
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 |
| Healthy volunteers/patients | — | — | — | 1 | — | — | — | — | 1 |
Indications Without Phase
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Ataxia | D001259 | — | R27.0 | — | — | — | — | 2 | 2 |
| Cerebellar ataxia | D002524 | — | — | — | — | — | — | 2 | 2 |
| Overweight | D050177 | — | E66.3 | — | — | — | — | 1 | 1 |
| Deglutition disorders | D003680 | — | R13.1 | — | — | — | — | 1 | 1 |
| Metabolic diseases | D008659 | EFO_0000589 | E88.9 | — | — | — | — | 1 | 1 |
| Lysosomal storage diseases | D016464 | — | — | — | — | — | — | 1 | 1 |
| Sarcopenia | D055948 | EFO_1000653 | M62.84 | — | — | — | — | 1 | 1 |
| Duchenne muscular dystrophy | D020388 | EFO_0000429 | — | — | — | — | — | 1 | 1 |
| Muscular dystrophies | D009136 | EFO_0000757 | G71.0 | — | — | — | — | 1 | 1 |
| Myalgia | D063806 | — | M79.1 | — | — | — | — | 1 | 1 |
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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 95 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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