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Original article
Cerliponase alfa, marketed as Brineura, is an enzyme replacement treatment for Batten disease, which is a form of neuronal ceroid lipofuscinosis.

On 27 April 2017, it was the approved by the United States Food and Drug Administration to slow loss of walking ability in symptomatic children over three years old with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). The disease is also known as tripeptidyl peptidase-1 (TPP1) deficiency. Also, it was approved by European Medicines Agency on 30 May 2017. In the United Kingdom NICE evaluated Cerliponase alfa for the treatment of CLN2 and deemed it not cost-effective.

It can be given by an intraventricular route which allows significant uptake into the brain.

It was developed by BioMarin Pharmaceutical.

Lead
Cerliponase alfa, marketed as Brineura, is an enzyme replacement treatment for Batten disease, a neurodegenerative lysosomal storage disease. Specifically, cerliponase alfa is meant to to slow loss of motor function in symptomatic children over three years old with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). The disease is also known as tripeptidyl peptidase-1 (TPP1) deficiency. Approved by the United States Food and Drug Administration on 27 April 2017, this is the first treatment for a neuronal ceroid lipofuscinosis of its kind, acting to slow disease progression rather than palliatively treat symptoms by giving patients the TPP1 enzyme they are lacking.

History
TPP1 was identified as the enzyme deficient in CLN2 batten disease in the late 1990's.


 * Discovery of TPP1 deficiency and gene
 * Clinical trials
 * FDA approval
 * Biomarin production
 * Other approval: Also, it was approved by European Medicines Agency on 30 May 2017. In the United Kingdom NICE evaluated Cerliponase alfa for the treatment of CLN2 and deemed it not cost-effective.
 * price?
 * more agencies

Structure and Biomolecular Mechanism

 * size
 * M6P targeting sequence and transport to lysosome
 * serine protease function and catalytic active site amino acids
 * cleaves N terminal tripeptide

Administration

 * intraventricular to cerebral spinal fluid
 * dosage/frequency of treatment