FDA Approves Expanded Use of BioMarin’s BRINEURA for CLN2

The US Food and Drug Administration (FDA) has broadened the approval of BioMarin Pharmaceutical’s enzyme replacement therapy, BRINEURA (cerliponase alfa), to include children younger than three years old with neuronal ceroid lipofuscinosis type 2 (CLN2) disease. This recent expansion extends the therapy's previous indication, which was initially approved to reduce the loss of ambulation in symptomatic children aged three and older with late infantile CLN2 disease. Now, BRINEURA is intended for children of all ages with CLN2, regardless of whether they are showing symptoms.

The supplemental biologics licence application (sBLA) for BRINEURA was backed by data from a Phase II clinical trial named Study 190-203. This open-label, multicentre trial involved children aged one to six years at the start and continued for approximately three years. The research indicated that the treatment effectively slowed the decline in motor function and delayed the disease's onset even in children younger than three. Additionally, the study found that the safety profile of BRINEURA in children under three was consistent with the existing safety data of the medication.

The results demonstrated that intraventricularly-administered BRINEURA significantly slowed the progression of CLN2 disease. Findings from the trial also suggested that starting treatment before the age of three could delay the disease's onset, offering a promising option for early intervention.

Hank Fuchs, the president of worldwide research and development at BioMarin Pharmaceutical, expressed that the expanded approval is a vital step in enabling children to start treatment with BRINEURA as early as possible. "The 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 the disease," Fuchs stated. He emphasized the urgency, noting that every day is crucial for families affected by serious genetic conditions like CLN2, which is known for its rapid onset of neurodegenerative symptoms. Fuchs added that BioMarin has been diligently working since BRINEURA’s initial approval to support its use in children of all ages, even before they exhibit symptoms.

In June 2023, another BioMarin product, Roctavian, received FDA approval as a one-time therapy for adults with severe haemophilia A, marking a significant achievement for the company in addressing genetic disorders.

This expanded approval of BRINEURA provides new hope for families and children battling CLN2 disease. By allowing treatment to begin at an earlier age, it opens up the possibility of mitigating the severe impacts of this rapid-onset neurodegenerative condition, potentially altering the disease's progression and improving the quality of life for affected children.