BRINEURA (cerliponase alfa), simple class
RARE DISEASE - New medicinal product
Opinions on drugs -
Posted on
Oct 24 2018
Reason for request
Inclusion
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.
Clinical Benefit
Substantial |
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Clinical Added Value
moderate |
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