cipaglucosidase alfa 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | BioActivity |

Stem definition	Drug id	CAS RN
enzymes	5721	2359727-71-0

Description:

Molecule	Description
Molfile Synonyms: cipaglucosidase alfa ATB200 pombiliti rhGAA-amicus recombinant human acid alpha-glucosidase enzyme-amicus	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. Molecular weight: Formula: CLOGP: LIPINSKI: None HAC: None HDO: None TPSA: ALOGS: ROTB: None

Molecule

Description

Molfile

Synonyms:

cipaglucosidase alfa
ATB200
pombiliti
rhGAA-amicus
recombinant human acid alpha-glucosidase enzyme-amicus

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.

Molecular weight:
Formula:
CLOGP:
LIPINSKI: None
HAC: None
HDO: None
TPSA:
ALOGS:
ROTB: None

Drug dosage:

None

ADMET properties:

None

Approvals:

Date	Agency	Company	Orphan
March 20, 2023	EMA	AMICUS THERAPEUTICS EUROPE LIMITED

FDA Adverse Event Reporting System (Female)

None

FDA Adverse Event Reporting System (Male)

None

FDA Adverse Event Reporting System (Geriatric)

None

FDA Adverse Event Reporting System (Pediatric)

None

Pharmacologic Action:

Source	Code	Description
ATC	A16AB23	ALIMENTARY TRACT AND METABOLISM OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS Enzymes

Drug Use | Suggest Off label Use Form| |View source of the data|

Disease	Relation	SNOMED_ID	DOID
Late-onset Pompe disease	indication	722343009

🐶 Veterinary Drug Use

None

🐶 Veterinary products

None

Acid dissociation constants calculated using MoKa v3.0.0

None

Orange Book patent data (new drug applications)

None

Orange Book exclusivity data (new drug applications)

None

Bioactivity Summary:

Target	Class	Pharos	UniProt	Action	Type	Activity value (-log[M])	Mechanism action	Bioact source	MoA source
Cation-independent mannose-6-phosphate receptor	Membrane receptor	P11717	MPRI_HUMAN	BINDING AGENT	Kd	9.05		DRUG LABEL	DRUG LABEL

External reference:

ID	Source
CHEMBL4594329	ChEMBL_ID
DB16708	DRUGBANK_ID
C5421138	UMLSCUI
11346	INN_ID
D11798	KEGG_DRUG
4SED7F4BSG	UNII

Pharmaceutical products:

None