Products
Cerliponase alfa was approved in the United States in 2017 as a solution for injection (Brineura). The drug’s extremely high price is controversial.
Structure and properties
Cerliponase alfa is a recombinant tripeptidyl peptidase 1 (rhTPP1) produced by biotechnological methods.
Effects
Cerliponase alfa replaces the insufficiently active enzyme tripeptidyl peptidase 1 (TPP1) in patients with the disease. Treatment may slow disease progression and stabilize patients.
Indications
For the treatment of late infantile neuronal ceroid lipofuscinosis (NCL2 disease/CLN2 disease). It is also referred to as pediatric dementia. This is a very rare and severe autosomal recessive, neurodegenerative lysosomal storage disease that occurs in children from about two to four years of age. It manifests in speech disorders, seizures, movement disorders, dementia, blindness, and eventually takes a fatal outcome at about eight to twelve years of age, among other symptoms.
Dosage
According to the SmPC. The drug is administered into the cerebrospinal fluid (intraventricular).
