U.S. FDA Approves BioMarin's BRINEURA For Children Under 3 Years With CLN2 Disease

Now Approved for Children of All Ages with CLN2 Batten Disease, Regardless of Whether They Yet Show Symptoms

SAN RAFAEL, Calif., July 24, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced that the U.S. Food and Drug Administration (FDA) has approved the company's supplemental Biologics License Application (sBLA) for BRINEURA® (cerliponase alfa) to slow the loss of ambulation in children of all ages with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as tripeptidyl peptidase 1 (TPP1) deficiency. Previously, BRINEURA was indicated in symptomatic children 3 years of age 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.