05:39 AM EDT, 07/25/2024 (MT Newswires) -- BioMarin Pharmaceutical ( BMRN ) said late Wednesday that the US Food and Drug Administration has approved its supplemental biologics license application for Brineura to slow the loss of ambulation in children of all ages with neuronal ceroid lipofuscinosis type 2, or CLN2, also known as tripeptidyl peptidase 1 deficiency.

Previously, Brineura, or cerliponase alfa, was indicated in symptomatic children three years and older with late infantile CLN2 disease.

This expanded indication now includes children of all ages with CLN2 disease, regardless of whether they are symptomatic or presymptomatic.

Data from a phase 2 multicenter trial over three years in children aged one to six at baseline, including eight children below three, supported the supplemental application. Brineura slowed the decline in motor function and delayed disease onset in children with CLN2 disease.

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