The U.S. Food and Drug Administration (FDA) has approved Brineura (cerliponase alfa) for the treatment of Neuronal ceroid lipofuscinosis 2 (CLN2) disease in children under three years of age.
CLN2 disease is a rare disorder that affects the nervous system. CLN2 is caused by changes in the TPP1 gene. All types of NCL also belong to a larger group of diseases known as lysosomal storage disorders. Children with CLN2 may experience:
- Speech delay
- Seizures that do not respond to medications
- Loss of muscle coordination
- Muscle twitches
- Loss of vision
- Developmental delay
- Intellectual disability
Cerliponase alfa is an enzyme replacement therapy that is an recombinant form of human TPP1. The treatment targets the restoration of the TPP1 enzyme and breaks down the storage materials that cause the disease.
The approval is based on results from Study 190-203 testing the treatment of CLN2 in children aged one to six years with cerliponase alfa, over three years. The trial was a phase 2, open-label, multicenter trial. From the data, a slowing in the decline of motor function and delayed disease onset was observed. The treatment also showed a well-tolerated safety profile.
For more information on rare neurological diseases, visit https://checkrare.com/diseases/neurology-nervous-system-diseases/