Cerliponase alfa 150mg/5ml solution for infusion vials
Requires a prescription from a doctor or prescriber
Official documents, adverse reaction reporting, and safety monitoring
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Official medicine documents
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Drug safety updates
MHRA alerts for Cerliponase alfa
Safety monitoring data
Yellow Card reports
The MHRA Yellow Card scheme collects reports of suspected side effects from healthcare professionals and patients. View the Drug Analysis Profile (iDAP) for real-world adverse reaction data.
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Suspected adverse reactions reported for Cerliponase alfa
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Data from the MHRA Yellow Card scheme. A reported reaction does not necessarily mean the medicine caused it. Contains public sector information licensed under the Open Government Licence v3.0.
EudraVigilance
The European Medicines Agency (EMA) collects suspected adverse reaction reports from across the EU/EEA through the EudraVigilance system. Search for safety data on this medicine.
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Suspected adverse reactions reported for Cerliponase alfa
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EudraVigilance data is published by the European Medicines Agency (EMA). A suspected adverse reaction is not necessarily caused by the medicine.
1 branded products available
MHRA licensed products
View all licensed products for Cerliponase alfa on the MHRA register
Brineura 150mg/5ml solution for infusion vials
Therapeutically similar medicines
Similarity is based on WHO Anatomical Therapeutic Chemical (ATC) classification and on a factual NHS dm+d therapeutic-grouping code prefix. Source data: NHS dm+d via TRUD (OGL v3.0), WHO ATC/DDD Index.
NHS prescribing volume and spending trends
Guidelines from the National Institute for Health and Care Excellence
NICE clinical guidance(1)
Source: National Institute for Health and Care Excellence (NICE). Contains public sector information licensed under the Open Government Licence v3.0.
Check stock at pharmacies and supply information
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Supply & safety information
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Pharmacy links redirect to the retailer's own search and do not represent real-time stock levels. Shortage and safety information sourced from MHRA drug safety updates (gov.uk, Crown Copyright under OGL v3.0).
Codes for healthcare professionals and prescribing systems
These codes are used by healthcare IT systems and prescribers to identify this medicine.
NHS UK identifiers
Browse tools
SNOMED CT and dm+d codes from NHS TRUD (Technology Reference data Update Distribution), licensed under the Open Government Licence v3.0. ATC codes from the WHO Collaborating Centre for Drug Statistics Methodology (whocc.no).
Active and completed clinical studies from ClinicalTrials.gov
Source: ClinicalTrials.gov, a database of the U.S. National Library of Medicine (NLM), National Institutes of Health (NIH). Data accessed via ClinicalTrials.gov API v2. Trial information is provided for research purposes and does not constitute medical advice.
Academic studies and reviews for this medicine's active substance
Showing the 50 most relevant studies.
Reviews & meta-analyses: 10 · Randomised trials: 14 · 1998–2026
Showing the 50 most relevant studies, sorted by most relevant.
J. M. Kirkwood, M. H. Strawderman, M. Ernstoff, et al.
Journal of Clinical Oncology, 2023
E. Van Cutsem, M. Tempero, D. Sigal, et al.
Journal of Clinical Oncology, 2020
H. Gisslinger, C. Klade, P. Georgiev, et al.
The Lancet. Haematology, 2020
U. Platzbecker, M. D. Della Porta, V. Santini, et al.
Lancet, 2023
M. Haynes, R. Giovanelli, B. Kent, et al.
The Astrophysical Journal, 2018
J. Díaz-Manera, P. Kishnani, H. Kushlaf, et al.
The Lancet. Neurology, 2021
B. Cho, J. Lee, Yi-long Wu, et al.
Journal of thoracic oncology : official publication of the International Association for the Study of Lung Cancer, 2023
T. Barbui, A. Vannucchi, V. De Stefano, et al.
The Lancet. Haematology, 2021
J. Kiladjian, C. Klade, P. Georgiev, et al.
Leukemia, 2022
Eric L. Wallace, O. Goker-Alpan, William R. Wilcox, et al.
Journal of Medical Genetics, 2023
Sources: aggregated from Europe PMC (EMBL-EBI), OpenAlex, Crossref, PubMed and other open scholarly databases. Retracted articles are excluded. Study information is provided for research purposes and does not constitute medical advice.
Pharmacology and chemical data from DrugBank
Key facts
Drug status
Approved
Major interactions
None known
Half-life
7.7 hours
Mechanism
CLN2 disease is a neurodegenerative disease associated with a variety of mutatio…
Food interactions
None known
Human targets
2 targets
Data: DrugBank · CC BY-NC 4.0
Pharmacokinetics at a glance
Absorption
300 mg
[L51319]
…
Half-life
300 mg
Volume of distribution
300 mg
Metabolism
[L51319]
Clearance
300 mg
Pharmacokinetic data: DrugBank · CC BY-NC 4.0
[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.
Cerliponase alfa (rhTTP1), a proenzyme, is taken up by target cells in the CNS and is translocated to the lysosomes through the Cation Independent Mannose-6-Phosphate Receptor (CI-MPR, also known as M6P/IGF2 receptor). Cerliponase alfa is activated in the lysosome and the activated proteolytic form of rhTPP1 cleaves tripeptides from the N-terminus of proteins.[A264354][A264359][L51319]
How the body processes this drug — absorption, distribution, metabolism, and elimination
[L51319]
Following intraventricular infusions of 300 mg of cerliponase alfa, the median maximum concentration (Cmax) in cerebrospinal fluid (CSF) was 1260, 1630 and 1390 ug/ml at day 1, week 5, and week 13, respectively. The median Tmax was 4.5, 4.3 and 4.3 h on day 1, week 5 and week 13, respectively. The area under the CSF concentration–time curve from 0 to the last measurable concentration (AUC0–t) 9290, 12,400 and 10,500 ug x h/ml, respectively.
[A264354][L51319]
[A264354][L51319]
[A264354][L51319]
[L51319]
[A264354][L51319]
Proteins and enzymes this drug interacts with in the body
PMID:18817523 PMID:2963003
Lysosomal enzymes bearing phosphomannosyl residues bind specifically to mannose-6-phosphate receptors in the Golgi apparatus and the resulting receptor-ligand complex is transported to an acidic prelysosomal compartment where the low pH mediates the dissociation of the complex .
PMID:18817523 PMID:2963003
The receptor is then recycled back to the Golgi for another round of trafficking through its binding to the retromer .
PMID:18817523
This receptor also binds IGF2 .
PMID:18046459
Acts as a positive regulator of T-cell coactivation by binding DPP4 PMID:10900005
PMID:11054422 PMID:19038966 PMID:19038967
May act as a non-specific lysosomal peptidase which generates tripeptides from the breakdown products produced by lysosomal proteinases .
PMID:11054422 PMID:19038966 PMID:19038967
Requires substrates with an unsubstituted N-terminus PMID:19038966
ATC A16AB17
Chemical identifiers
CAS, UNII, InChI Key and database cross-references
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Chemical identifiers
CAS, UNII, InChI Key and database cross-references
Linked compound data from DrugBank Open Data (CC BY-NC 4.0)
Cerliponase alfa
Additional database identifiers
Drugs Product Database (DPD)
23028
HUGO Gene Nomenclature Committee (HGNC)
HGNC:5467
GeneCards
IGF2R
GenBank Gene Database
Y00285
GenBank Protein Database
33055
UniProt Accession
MPRI_HUMAN
HUGO Gene Nomenclature Committee (HGNC)
HGNC:2073
GeneCards
TPP1
UniProt Accession
TPP1_HUMAN
DrugBank citations
If you use DrugBank data in your research, please cite:
- DrugBank 6.02024Recommended citationKnox C., Wilson M., Klinger C.M., et alDrugBank 6.0: the DrugBank Knowledgebase for 2024Nucleic Acids Res. 2024 Jan 552(D1):D1265-D1275
- DrugBank 5.02018Wishart D.S., Feunang Y.D., Guo A.C., et alDrugBank 5.0: a major update to the DrugBank database for 2018Nucleic Acids Res. 2017 Nov 846(D1):D1074-D1082
- DrugBank 4.02014Law V., Knox C., Djoumbou Y., et alDrugBank 4.0: shedding new light on drug metabolismNucleic Acids Res. 2014 Jan 142(1):D1091-7
- DrugBank 3.02011Knox C., Law V., Jewison T., et alDrugBank 3.0: a comprehensive resource for 'omics' research on drugsNucleic Acids Res. 2011 Jan39(Database issue):D1035-41
- DrugBank 2.02008Wishart D.S., Knox C., Guo A.C., et alDrugBank: a knowledgebase for drugs, drug actions and drug targets.Nucleic Acids Research2008 Jan36(Database issue):D901-6
- DrugBank 1.02006Wishart D.S., Knox C., Guo A.C., et alDrugBank: a comprehensive resource for in silico drug discovery and exploration.Nucleic Acids Research2006 Jan 134(Database issue):D668-72