FDA Expands Indication for Brineura To Treat CLN2 Disease

The Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) for Brineura (cerliponase alfa; BioMarin Pharmaceuticals, San Rafael, CA), expanding the medication’s indication to be used as a treatment to slow the loss of ambulation in all children with neuronal ceroid lipofuscinosis type 2 (CLN2) disease. Previously, Brineura was indicated to treat only symptomatic children aged 3 years and older with late infantile CLN2 disease.

The sBLA approval is based on data from a phase 2, open-label, multicenter clinical trial (NCT02678689), which evaluated the safety, tolerability, and efficacy of Brineura for the treatment of children aged 1-6 years at baseline, including 8 children under age 3. Researchers assessed participants’ decline in motor function on the CLN2 Clinical Rating Scale. Of the treated children, 7 were matched to untreated children from a natural history cohort (n=18) as a comparator.

At final assessment (week 169):

• None of the children (0%) showed a 2-point decline, nor a score of 0, per the CLN2 Clinical Rating Scale.
• Of the matched natural history comparators, 11 children (61%) demonstrated an unreversed 2-point decline or score of 0 per the CLN2 Clinical Rating Scale.

The findings suggest that treatment with Brineura initiated before age 3 may delay disease onset as compared with untreated children. Brineura showed a safety profile in this patient population consistent with the previously characterized safety profile of the medication.

"Today's approval represents a significant step forward in enabling children to be treated with BRINEURA as early as possible, when we can have the greatest impact in altering the natural course of disease," said Hank Fuchs, MD, President of Worldwide Research and Development at BioMarin Pharmaceuticals. "We know that every day counts for families affected by serious genetic conditions such as CLN2 disease, which is characterized by a rapid onset of neurodegenerative symptoms. We have been working diligently since BRINEURA's initial approval to support this expanded use in children of all ages, even before they begin to show symptoms."