Brineura™ (cerliponase alfa) is the only treatment that directly addresses the cause of CLN2 disease by replacing the TPP1 enzyme
Brineura® (cerliponase alfa) is a prescription medication used to slow loss of ability to walk or crawl (ambulation) in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency.
CLN2 disease is a type of lysosomal storage disorder that affects cells in the brain. Lysosomes are in every cell and contain enzymes that break down materials in the cell. One of these enzymes is called TPP1, which is missingor not working properly in children with CLN2 disease.1
How is Brineura administered to my child?
Brineura is a type of treatment called enzyme replacement therapy (ERT). It’s administered through intraventricular infusion—a method that allows Brineura to be directly delivered into the fluid surrounding the brain, known as the cerebrospinal fluid.4
Before starting Brineura, your child will need to have an intraventricular access device surgically implanted just below their scalp.4 This is an established procedure in pediatric neurology,5 and allows direct delivery of Brineura into a ventricle in the brain. The access device is about the size of a penny. It’s recommended that the first dose of Brineura treatment begin at least 5 to 7 days after the access device is implanted.4
Brineura treatments will take about 4.5 hours every other week.4 You’ll work with your healthcare team to schedule treatments.
Your healthcare team will let you know how to prepare your child for infusion, and what to expect during this procedure. Your child will be monitored before, during, and after the infusion, and may receive medications to reduce the risk of infection.
Matty’s infusion day
Matty is a patient with CLN2 disease.
He's been on treatment with Brineura since 2015.
“After the first few infusions we became more comfortable, because it simply became routine for us. The initial surgery for the port was scary, so we felt like that was the first hurdle to jump. Once Matty had a few infusions, we became more comfortable with the process.”
—Joe, Matty’s dad
Learn how Brineura helps children with CLN2 disease
Brineura was evaluated in 24 children with CLN2 disease in a clinical study.
References: 1. Mole SE, Williams RE, Goebel HH. Correlations between genotype, ultrastructural morphology and clinical phenotype in the neuronal ceroid lipofus cinoses. Neurogenetics. 2005;6:107-126. 2. Haltia M. The neuronal ceroid-lipofuscinoses: from past to present. Biochimica et Biophysica Acta. 2006;1762:850-856. 3. Schulz A, Kohlschütter A, Mink J, Simonati A, Williams R. NCL diseases – clinical perspectives. Biochim Biophys Acta. 2013;1832:1801-1806. 4. Brineura [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2017. 5. Cohen-Pfeffer JL, Gururangan S, Lester T, et al. Intracerebroventricular delivery as a safe, long-term route of drug administration. Pediatr Neurol. 2017;67:23-35.