US FDA Nods BioMarin's Brineura For Children Under 3 years

BioMarin Pharmaceutical Inc. declared that the FDA has given the green light to the company's sBLA for Brineura to help delay the loss of walking ability in children with CLN2 disease, also known as TPP1 deficiency. Before, Brineura was approved for symptomatic children aged 3 and above with late infantile CLN2 disease. This new approval now covers all children with CLN2 disease, regardless of their symptoms or lack thereof.

"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."

Data from Study 190-203, a phase 2 clinical trial, backing the sBLA. The trial assessed Brineura treatment for around three years in kids aged 1-6 at baseline, with eight children under 3 years old.