EU CHMP recommends approval of Brineura (cerliponase alfa) for Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) disease- BioMarin

The Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), has adopted a positive opinion for the Marketing Authorization Application (MAA) for Brineura (cerliponase alfa), from BioMarin, to treat children with Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) disease, a form of Batten disease, which is also known as tripeptidyl peptidase 1 (TPP1) deficiency.

The CHMP positive opinion was adopted following an accelerated review procedure, reserved for medicinal products expected to be of major public health interest. The EMA revised process for accelerated assessment came into effect June 1, 2016, and Brineura is one of the first therapies to go through this process

The Brineura MAA was based on an open-label, dose-escalation study for Brineura in 24 patients with CLN2 disease between 3 and 8 years of age, as well as an open-label extension study. The primary objectives were to evaluate the safety and tolerability of intracerebroventricular-administered Brineura and to evaluate effectiveness using a CLN2 disease-specific rating scale score in comparison with natural history data after 48 and 72 weeks of treatment. In 2016, the CHMP accepted BioMarin's request for accelerated assessment. The EMA previously granted Brineura Orphan Drug Designation.