BRINEURA (cerliponase alfa), simple class

High clinical benefit for neuronal ceroid lipofuscinosis type 2 and moderate clinical added value in the management of this disease. 

• BRINEURA has been granted marketing authorisation for the treatment of neuronal ceroid lipofuscinosis type 2 (NCL2) or tripeptidyl peptidase-1 (TPP1) deficiency. 

• The efficacy data of a non-comparative open-label study demonstrate slowing of disease progression, based on a score evaluating 2 of its components: mobility and language, after 96 weeks of treatment, with no mortality data. These results, contrasted with those of a cohort of untreated patients, suggest a benefit of cerliponase alfa on the motor-language score.

• There are some uncertainties concerning its safety, associated with its intracerebroventricular route of administration and long-term efficacy.