Slovakia-Banská Bystrica: Medicinal products for the alimentary tract and metabolism

Brineura 150 mg solution for infusion (SUKL 4681C) contains the active substance alpha cerliponase, which belongs to a group of medicines known as substitution enzyme therapies.
Brineura (Cerliponasis alpha) is the first approved treatment for any form of Batten’s disease and is indicated to slow down the progression of the disease in paediatric symptomatic patients aged 3 years and older with late infantile neuronal ceroid lipofuscinose type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency. CLN2 is an extremely rare and fast-growing brain disorder affecting around 20 children born in the United States per year, i.e. less than 1 incidence of disease per million Americans.
Brineura is the first enzyme substitution treatment that is given directly to the fluid in the brain that treats the root cause of CLN2 by helping to replace the missing TPP1 enzyme in affected children.
It is used to treat patients with neuronal ceroid lipfuscinosis type 2 (CLN2), also known as Tripeptidylpeptidase 1 deficit (TPP1). CLN2 is a rare genetic disorder affecting children.
People with CLN2 do not have an enzyme called TPP1 or have too little, causing the accumulation of substances called lysosome accumulating agents. In people with CLN2, these substances accumulate in some parts of the body, especially in the brain.
This medicine replaces the missing enzyme called TPPA and thereby minimises the accumulation of lysosomal accumulation agents. This medicine works by slowing down the progress of the disease.
The recommended dose is 300 mg of cerliponase alpha, given once every 2 weeks by intracerebroventricular infusion, which means that it is administered to the brain brain by infusion through a surgically implanted container and catheter.

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