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BRINEURA® (cerliponase alfa)

BRINEURA® (cerliponase alfa) is the first and only product to treat CLN2 disease.1,2

 

 

What is BRINEURA®?

BRINEURA® (cerliponase alfa) is an enzyme replacement for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency.1 As the first and only product to treat CLN2 disease, BRINEURA® injection for intraventricular use must only be administered by a trained healthcare professional knowledgeable in intracerebroventricular administration in a healthcare setting.1

Mechanism of action

Cerliponase alfa is a recombinant form of human tripeptidyl peptidase-1 (rhTPP1). Cerliponase alfa is the inactive proenzyme (zymogen) form of a protease that is activated in the lysosome. Due to its attached mannose 6-phosphate residues, cerliponase alfa is bound by the cation-independent mannose-6-phosphate receptor (M6P, also known as the IGF2 receptor) on target cells and, following clathrin-mediated endocytosis of the complex, is translocated to lysosomes. Cerliponase alfac cleaves the lysosomal tripeptides and helps clear accumulation in the cell.1

For details of the frequency of adverse reactions, please refer to BRINEURA® Product Information.

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BRINEURA®

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References:

  1. Australian Product Information-CERLIPONASE ALFA (BRINEURA) 30 MG/ML SOLUTION FOR INJECTION. https://www.biomarin.com/en-au/brineura-product-information/. Accessed March 2023.
  2. FDA approves first treatment for a form of Batten disease. https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-form-batten-disease. Accessed March 2023.