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.