Cerliponase Alfa: First Global Approval

Abstract

Cerliponase alfa (Brineura™) is a recombinant human tripeptidyl peptidase-1 (TTP1) being developed by BioMarin Pharmaceutical Inc. for use in patients with late infantile 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 for the treatment of motor function loss in paediatric patients ≥3 years of age with CLN2.

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