The FDA has approved BioMarin’s supplemental BLA for Brineura (cerliponase alfa), expanding its use to children of all ages with neuronal ceroid lipofuscinosis type 2 (CLN2) disease.
CLN2 disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency, typically manifests with seizures and rapid neurodegeneration, leading to loss of motor and cognitive functions and often death by early adolescence.
Previously, Brineura was indicated only for symptomatic children aged three and older. The new approval includes both symptomatic and presymptomatic children, potentially allowing for earlier intervention in this rare, neurodegenerative condition.
The approval was supported by data from Study 190-203, which evaluated Brineura over three years in children aged one to six, including those under three years old. Results showed that the drug slowed the decline in motor function and delayed disease onset in these younger children.
Brineura, first approved in 2017, is an enzyme replacement therapy designed to restore TPP1 activity in the brain and central nervous system. It is the first and only approved treatment for CLN2 disease, administered intraventricularly to deliver the enzyme directly to the brain.
