Cerliponase Alfa: First Global Approval
Abstract
Cerliponase alfa (Brineura™) is a recombinant human tripeptidyl peptidase-1 (TPP1) being developed by BioMarin Pharmaceutical Inc. for use in patients with neuronal ceroid lipofuscinosis type 2 (CLN2), a paediatric neurodegenerative disease caused by a deficiency in TPP1. CLN2 is characterised by progressive impairment of motor function, language deficiencies, seizures, ataxia, blindness and early death, and intracerebroventricular infusion of cerliponase alfa has been shown to reduce the progression of functional decline. This article summarizes the milestones in the development of cerliponase alfa leading to its first global approval in the USA for the treatment of motor function loss in paediatric patients ≥3 years of age with CLN2, and subsequent approval in the EU for CLN2 in all ages.
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Several approaches to circumvent the blood-brain barrier have been utilized clinically: intracerebroventricular Cerliponase for Batten's disease, intrathecal Spinraza for infantile spinal muscular atrophy, intrathecal Ziconotide for chronic pain, and intranasal insulin, leptin, and oxytocin.
“These include intracerebroventricular administration of Cerliponase for Batten's disease (395), intrathecal administration of antisense oligo Spinraza targeting the survival motor neuron 1 gene (SMN1) for infantile spinal muscular atrophy (150), intrathecal Ziconited peptide for chronic pain (398), and intranasal administration of insulin, leptin, and oxytocin (103).”
From Blood-Brain Barrier: From Physiology to Disease and Back (2019) · cites this paper as [395]
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Blood-Brain Barrier: From Physiology to Disease and Back
Sweeney, M. D., Zhao, Z., Montagne, A., Nelson, A. R., & Zlokovic, B. V. (2019) · Physiological Reviews · doi:10.1152/physrev.00050.2017 · Cited by 2,294 · see citing works
Sondhi, D., Kaminsky, S. M., Hackett, N. R., Pagovich, O. E., Rosenberg, J. B., De, B. P., Chen, A., Van de Graaf, B., Mezey, J. G., Mammen, G. W., et al. (2020) · Science Translational Medicine · doi:10.1126/scitranslmed.abb5413 · Cited by 72 · see citing works
- Authors
- Markham, A.
- Corresponding author
- Markham, Anthony
- Venue
- Drugs
- Published
- 2017-06-06
- Volume
- 77
- Issue
- 11
- Pages
- 1247-1249
- ISSN
- 0012-6667 (ISSN-L)
- 1179-1950
- OpenAlex
- W2622228475
- Cited by
- 120
- References
- 0
- Citations by year
- 2026: 3 · 2025: 10 · 2024: 5 · 2023: 9 · 2022: 15 · 2021: 26 · 2020: 21 · 2019: 11 · 2018: 17 · 2017: 2 · 2015: 1
- FWCI
- 10.36
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Drugs (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Health Sciences › Medicine › Physiology › Lysosomal Storage Disorders Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Clinical Biochemistry › Metabolism and Genetic Disorders
- Health Sciences › Medicine › Rheumatology › Glycogen Storage Diseases and Myoclonus
- Keywords
- cerliponase alfa, tripeptidyl peptidase 1, neuronal ceroid lipofuscinosis type 2, CLN2, recombinant enzyme replacement therapy, intracerebroventricular infusion, motor function loss, drug approval
- MeSH terms
- Tripeptidyl-Peptidase 1, Child, Child, Preschool, Dipeptidyl-Peptidases and Tripeptidyl-Peptidases, Europe, Female, Humans, Male, Neuronal Ceroid-Lipofuscinoses, Recombinant Proteins, United States, United States Food and Drug Administration, Drug Approval, Disease Progression, Enzyme Replacement Therapy
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