Ultra Orphan Assessment

The Scottish Medicines Consortium (SMC) has completed its initial assessment of the evidence for this medicine using the ultra-orphan framework

cerliponase alfa (Brineura)

Indication under review: for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency.

Key points:

  • CLN2 disease is a severe, neurodegenerative condition, diagnosed in childhood with devastating symptoms affecting multiple aspects of the child’s life. There are no other medicines licensed for this condition.
  • A phase I/II study reported a clinically relevant treatment effect with cerliponase alfa, measured by the CLN2 motor/language (ML) scale at 48 weeks. This treatment effect was maintained through to week 96 in an extension study. Cerliponase alfa was also associated with significant treatment benefits when indirectly compared to standard of care from a historical control group.
  • The quality of life data are potentially difficult to interpret but can be considered positive. The stabilisation observed may be beneficial considering the decline in quality of life typically observed with CLN2 disease.
  • A model-based health economic evaluation suggests that cerliponase alfa is associated with a substantial gain in quality-adjusted life years compared to standard of care. However, the following issues add to the uncertainty of the results: assumptions regarding long term disease stabilisation; the distribution of patients in different starting health states; utility value estimates and the long time horizon.
  • Despite a Patient Access Scheme (PAS) that improves the cost-effectiveness of cerliponase alfa, the treatment’s cost in relation to its health benefits remains high.

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

Medicine details

Medicine name:
cerliponase alfa (Brineura)
SMC ID:
SMC2286
Indication:

For the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency.

Pharmaceutical company
BioMarin
BNF chapter
Central nervous system
Submission type
Ultra-orphan initial assessment
Date Published
12 October 2020
Additional notes

A new approach for assessing medicines for extremely rare conditions started in October 2018. Further information is available on our ultra-orphan webpage and in the Scottish Government’s guidance.