BioMarin's BRINEURA Receives FDA Approval for All Ages with CLN2 Disease

BioMarin Pharmaceutical has announced that the U.S. Food and Drug Administration (FDA) has expanded the approval of its drug, BRINEURA (cerliponase alfa), to treat children of all ages with CLN2 disease, also known as Batten disease. Previously, the drug was only approved for symptomatic children aged 3 and above.

This expanded approval is based on a clinical trial showing that BRINEURA can slow the progression of the disease, even in children under 3 years old who may not yet show symptoms. The drug is administered directly into the cerebrospinal fluid and works by replacing a missing enzyme.

CLN2 disease is a rare genetic disorder that causes progressive damage to the brain and nervous system. It typically begins with seizures and language delays and eventually leads to blindness, loss of motor function, and death. There is currently no cure for the disease.