Cerliponase Alfa: First Global Approval

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Funding: The preparation of this review was not supported by any external funding.

Conflicts of interest: A. Markham is a contracted employee of Adis/Springer SBM, is responsible for the article content and declares no relevant conflicts of interest.

Additional information about this Adis Drug Review can be found here.

Abstract

Cerliponase alfa (Brineura™) is a recombinant human tripeptidyl peptidase-1 (rhTPP1) 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 (ICV) 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 this first global approval for the treatment of motor function loss in paediatric patients ≥3 years of age with CLN2. Access to the full article can be found here.

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