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U.S. Food and Drug Administration Approves BioMarin's BRINEURA® (cerliponase alfa) 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/ --
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"Now Approved for Children of All Ages with CLN2 Batten Disease, Regardless of Whether They Yet Show Symptoms\nSAN 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.\n\n \n \n \n \n \n \n\n \n\"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, M.D., president of Worldwide Research and Development at BioMarin. \"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.\"\nThe sBLA is supported by data from Study 190-203, a Phase 2, open-label, multicenter trial evaluating BRINEURA treatment over the span of approximately three years in children aged 1-6 years at baseline, including eight children less than 3 years of age. Results from Study 190-203, which were presented at the 20th Annual We're Organizing Research on Lysosomal Diseases meeting (WORLDSymposium) in February, showed that intraventricular (intracerebroventricular, ICV)-administered BRINEURA slowed the decline in motor function and delayed disease onset in children with CLN2 disease, including those who were under 3 years of age. BRINEURA's safety profile has been well-characterized, and safety results in children under 3 years of age were similar to the known safety profile of the medicine. In addition to confirming that treatment initiated after 3 years of age significantly slows the progress...