PPIDT00370

Drug Information
NameCerliponase alfa
SequenceSYSPEPDQRRTLPPGWVSLGRADPEEELSLTFALRQQNVERLSELVQAVSDPSSPQYGKYLTLENVADLVRPSPLTLHTVQKWLLAAGAQKCHSVITQDFLTCWLSIRQAELLLPGAEFHHYVGGPTETHVVRSPHPYQLPQALAPHVDFVGGLHRFPPTSSLRQRPEPQVTGTVGLHLGVTPSVIRKRYNLTSQDVGSGTSNNSQACAQFLEQYFHDSDLAQFMRLFGGNFAHQASVARVVGQQGRGRAGIEASLDVQYLMSAGANISTWVYSSPGRHEGQEPFLQWLMLLSNESALPHVHTVSYGDDEDSLSSAYIQRVNTELMKAAARGLTLLFASGDSGAGCWSVSGRHQFRPTFPASSPYVTTVGGTSFQEPFLITNEIVDYISGGGFSNVFPRPSYQEEAVTKFLSSSPHLPPSSYFNASGRAYPDVAALSDGYWVVSNRVPIPWVSGTSASTPVFGGILSLINEHRILSGRPPLGFLNPRLYQQHGAGLFDVTRGCHESCLDEEVEGQGFCSGPGWDPVTGWGTPNFPALLKTLLNP
DrugBank_IDDB13173
Typebiotech
IndicationCerliponase alfa is indicated to slow the loss of ambulation in pediatric patients with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as tripeptidyl peptidase 1 (TPP1) deficiency.[L51319, L51324, L51329] It is approved for use in children in the US, whereas in Europe and Canada, it is approved for use in patients of all ages.

Dosage Forms
Form Route Strength
Injection, solution Intracerebral
150 mg
Injection, solution Intracerebral; Parenteral
150 MG
Injection, solution; kit Intraventricular
150 mg/5mL
Solution Intracerebral
150 mg / 5 mL
Solution Intracerebral
3000000 mg
Target Information
Role Uniprot_ID Gene_Name Entity_Name Organism Actions Internal link
target P11717 IGF2R Cation-independent mannose-6-phosphate receptor Homo sapiens ligand Link