What condition is Brineura used to treat?
Brineura (cerliponase alfa) is used to slow the loss of ambulation (ability to crawl or walk unaided) in patients with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency. Brineura is used in pediatric patients 3 years of age and over who have symptomatic disease.
CLN2 is a neurodegenerative disease and a type of Batten disease. It is caused by a lack of the enzyme TPP1, which leads to the build up of lysosomal storage materials in the central nervous system (CNS) and results in a progressive decline in motor function.
Brineura is a proenzyme of TPP1. It is taken up by the CNS and is activated in the lysosomes, which results in their break down.
- Food and Drug Administration (FDA). Brineura. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/761052s008lbl.pdf. [Accessed March 9, 2021].