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| Stem definition | Drug id | CAS RN |
|---|---|---|
| enzymes | 5721 | 2359727-71-0 |
| Molecule | Description |
|---|---|
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Synonyms:
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Pombiliti (cipaglucosidase alfa) is a long-term enzyme replacement therapy used in combination with the enzyme stabiliser miglustat for the treatment of adults with late-onset Pompe disease (acid alpha-glucosidase [GAA] deficiency). Cipaglucosidase alfa is intended to replace the absent or impaired endogenous enzyme. Cipaglucosidase alfa is stabilised by miglustat minimising the loss of enzyme activity in the blood during infusion of this hydrolytic glycogen-specific enzyme enriched with bis-M6P N-glycans for high affinity cation-independent mannose-6-phosphate receptor (CI-MPR) binding. After binding, it is internalised in the lysosome where it undergoes proteolytic cleavage and N-glycan trimming which are both required to yield the most mature and active form of the GAA enzyme. Cipaglucosidase alfa then exerts enzymatic activity in cleaving glycogen and reducing intramuscular glycogen, and ameliorating tissue damage.
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| Date | Agency | Company | Orphan |
|---|---|---|---|
| March 20, 2023 | EMA | AMICUS THERAPEUTICS EUROPE LIMITED | |
| Sept. 28, 2023 | FDA | AMICUS THERAP US |
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| Source | Code | Description |
|---|---|---|
| ATC | A16AB23 | ALIMENTARY TRACT AND METABOLISM OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS Enzymes |
| Disease | Relation | SNOMED_ID | DOID |
|---|---|---|---|
| Late-onset Pompe disease | indication | 722343009 |
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| Target | Class | Pharos | UniProt | Action | Type | Activity value (-log[M]) | Mechanism action | Bioact source | MoA source |
|---|---|---|---|---|---|---|---|---|---|
| Cation-independent mannose-6-phosphate receptor | Membrane receptor | BINDING AGENT | Kd | 9.05 | DRUG LABEL | DRUG LABEL |
| ID | Source |
|---|---|
| CHEBI:757827 | CHEBI |
| CHEMBL4594329 | ChEMBL_ID |
| C000731755 | MESH_SUPPLEMENTAL_RECORD_UI |
| DB16708 | DRUGBANK_ID |
| 1025331000122100 | SNOMEDCT_US |
| 4042645 | VANDF |
| 4042647 | VANDF |
| C5421138 | UMLSCUI |
| 11346 | INN_ID |
| 371786 | MMSL |
| 42028 | MMSL |
| d10116 | MMSL |
| 019568 | NDDF |
| D11798 | KEGG_DRUG |
| 4SED7F4BSG | UNII |
| 2667433 | RXNORM |
| Product | Category | Ingredients | NDC | Form | Quantity | Route | Marketing | Label |
|---|---|---|---|---|---|---|---|---|
| POMBILITIATGA | HUMAN PRESCRIPTION DRUG LABEL | 1 | 71904-200 | INJECTION, POWDER, LYOPHILIZED, FOR SOLUTION | 105 mg | INTRAVENOUS | BLA | 27 sections |