First Global Approved Drug-Recombinant Human Tripeptidyl Peptidase-1 (TPP1) for the Treatment of CLN2 Disease

Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), a paediatric neurodegenerative disease caused by a deficiency in TPP1 is characterised by progressive impairment of motor function, language deficiencies, seizures, ataxia, blindness and early death. Cerliponase alfa (Brineura) is the first global approved drug-recombinant human tripeptidyl peptidase-1 (TPP1) for treating CLN2, and it is a recombinant human tripeptidyl peptidase-1 (TPP1) being developed by BioMarin Pharmaceutical Inc. This article summarizes the milestones in the development of cerliponase alfa from the aspects of chemistry, patent status, market sale value, mechanism, pharmacokinetics, and the effect and safety data obtained from clinical trials. Then the important information of cerliponase alfa will be provided for domestic researcher to further understand the drug, and may provide some help for the research and treatment of relevant rare disease.