Scottish Medicines Consortium completes initial assessment of evidence for cerliponase alfa (Brineura) for treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease – using the ultra-orphan framework

Assessment includes a phase I/II study that reported a clinically relevant treatment effect with cerliponase alfa, measured by the CLN2 motor/language scale at 48 weeks; and this treatment effect was maintained through to week 96 in an extension study.

SPS commentary:

The Scottish Government Medicines Policy Branch will notify Health Boards when this medicine is available for prescribing within the ultra-orphan pathway.  In the meantime, any requests to access treatment should be considered through local non-formulary processes.

Scottish Medicines Consortium