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

Stem definition	Drug id	CAS RN
enzymes	5230	151662-36-1

Description:

Molecule	Description
Synonyms: cerliponase alfa rhTTP1 brineura	Cerliponase alfa is a purified human enzyme produced by recombinant DNA technology in a Chinese hamster ovary cell line. The active substance is a recombinant human tripeptidyl peptidase-1 (rhTPP1), a lysosomal exopeptidase. The primary activity of the mature enzyme is the cleavage of N-terminal tripeptides from a broad range of protein substrates. CLN2 disease is a neurodegenerative disease caused by deficiency of the lysosomal enzyme tripeptidyl peptidase-1 (TPP1), which catabolizes polypeptides in the CNS. Deficiency in TPP1 activity results in the accumulation of lysosomal storage materials normally metabolized by this enzyme in the central nervous system (CNS), leading to progressive decline in motor function. Molecular weight: Formula: CLOGP: LIPINSKI: None HAC: None HDO: None TPSA: ALOGS: ROTB: None

Molecule

Description

Synonyms:

cerliponase alfa
rhTTP1
brineura

Cerliponase alfa is a purified human enzyme produced by recombinant DNA technology in a Chinese hamster ovary cell line. The active substance is a recombinant human tripeptidyl peptidase-1 (rhTPP1), a lysosomal exopeptidase. The primary activity of the mature enzyme is the cleavage of N-terminal tripeptides from a broad range of protein substrates. CLN2 disease is a neurodegenerative disease caused by deficiency of the lysosomal enzyme tripeptidyl peptidase-1 (TPP1), which catabolizes polypeptides in the CNS. Deficiency in TPP1 activity results in the accumulation of lysosomal storage materials normally metabolized by this enzyme in the central nervous system (CNS), leading to progressive decline in motor function.

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

Drug dosage:

Dose	Unit	Route
21	mg	P

ADMET properties:

None

Approvals:

Date	Agency	Company
May 30, 2017	EMA	BioMarin International Limited
April 27, 2017	FDA	BIOMARIN PHARM
Sept. 20, 2019	PMDA	BioMarin Pharmaceutical Japan K.K.

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	A16AB17	ALIMENTARY TRACT AND METABOLISM OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS Enzymes
FDA CS	M0534354	Dipeptidyl-Peptidases and Tripeptidyl-Peptidases
FDA EPC	N0000193414	Hydrolytic Lysosomal N-terminal Tripeptidyl Peptidase

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

Disease	Relation	SNOMED_ID	DOID
Late-infantile neuronal ceroid lipofuscinosis	indication	14637005

🐶 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:

None

External reference:

ID	Source
X8R2D92QP1	UNII
4036691	VANDF
C4475875	UMLSCUI
CHEMBL3544921	ChEMBL_ID
DB13173	DRUGBANK_ID
D10813	KEGG_DRUG
9970	INN_ID
256784	MMSL
32694	MMSL
d08579	MMSL
017191	NDDF
734039005	SNOMEDCT_US
763608006	SNOMEDCT_US
1922436	RXNORM
C000621431	MESH_SUPPLEMENTAL_RECORD_UI

Pharmaceutical products:

None