cerliponase alfa

Cerliponase alfa is a recombinant human enzyme used as an enzyme replacement therapy for neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency or a form of Batten disease. CLN2 is a rare pediatric-onset neurodegenerative lysosomal storage disorder caused by deficiency of the lysosomal enzyme TPP1. Cerliponase alfa is administered via intraventricular infusion directly into the cerebrospinal fluid to deliver TPP1 to the central nervous system. The drug works by replacing deficient TPP1 in affected patients; once inside target cells, it binds to mannose-6-phosphate receptors and is transported to lysosomes where it becomes activated and cleaves N-terminal tripeptides from protein substrates. This slows progressive loss of motor function in children with CLN2 but does not cure the underlying disease. Developers: BioMarin Manufacturers: BioMarin

Brand names

Brineura

Other names

recombinant human tripeptidyl peptidase 1rhTPP1rhTPP-1rhTPP 1