BioMarin’s Brineura Receives Significant Label Expansion from FDA for Children Under 3 with CLN2 Disease

1. Label Expansion: The FDA has approved BioMarin's supplemental biologics license application (sBLA) for Brineura (cerliponase alfa), expanding its indication to include children under the age of three with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease.
2. Previous Indication: Brineura was initially approved for symptomatic children aged three and older with late infantile CLN2 disease to slow the loss of ambulation.
3. Phase II Study: The sBLA was supported by a Phase II study (Study 190-203) that evaluated Brineura in children aged one to six years over three years, demonstrating its ability to decelerate motor ability deterioration and postpone disease onset, even in very young children.
4. Administration: Brineura is administered via intraventricular injection and has shown an established safety profile in children under three years old.
5. Impact: This expanded approval allows children of all age groups with CLN2 disease to receive Brineura, potentially delaying the onset of neurodegenerative symptoms.