BioMarin secures broader approval for CLN2 disease therapy

The FDA has recently given approval to BioMarin's supplemental Biologics License Application (BLA) for Brineura (cerliponase alfa). This approval extends the drug's use to include children of all ages afflicted with neuronal ceroid lipofuscinosis type 2 (CLN2) disease. This expansion marks a significant advancement, as CLN2 disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency, typically manifests with severe symptoms such as seizures and rapid neurodegeneration. These symptoms typically result in the loss of motor and cognitive abilities and often lead to early death during adolescence.

Before this new approval, Brineura was only indicated for treatment in symptomatic children aged three years and older. With the recent decision by the FDA, both symptomatic and presymptomatic children can now receive Brineura, enabling potentially earlier intervention for this debilitating and rare neurodegenerative disease. The approval signifies an important milestone, as early treatment could be crucial in managing and possibly mitigating the disease's progression.

The decision to extend Brineura's use was based on data derived from Study 190-203, which investigated the drug's efficacy over a three-year period in children aged one to six years, including those under the age of three. Findings from this study revealed that Brineura was effective in slowing down the deterioration of motor functions and delaying the onset of the disease in these younger age groups. These promising results underscore the potential benefits of administering Brineura at an earlier stage of CLN2 disease.

Initially approved in 2017, Brineura is an enzyme replacement therapy specifically designed to restore the activity of TPP1 in the brain and central nervous system. It stands as the first and only treatment approved for CLN2 disease, highlighting its significance in the realm of rare neurological conditions. The drug is administered intraventricularly, ensuring that the enzyme is delivered directly to the brain, which is essential for addressing the neurological aspects of the disease.

In summary, the FDA's expanded approval of Brineura offers new hope for children of all ages who suffer from CLN2 disease. By potentially enabling earlier intervention, this approval could play a critical role in improving the quality of life and extending the lifespan of those affected by this severe condition. The supporting data from extensive studies affirm the drug's capability to slow disease progression, marking a notable achievement in the treatment of neurodegenerative diseases.