BRINEURA 150 mg (cerliponase alfa) - ) Rare disease

The Committee deems that the clinical benefit of BRINEURA 150 mg (cerliponase alfa) solution for infusion remains substantial in the MA indication.

Considering:

• limited initial efficacy data from a non-comparative study, demonstrating a slowdown in disease progression based on a score assessing two components, i.e. motricity and language, after a maximum of 96 weeks of treatment, with no mortality data; with comparison of the results with those from an untreated patient cohort suggesting a benefit of treatment for the motricity/language score,
• new data from three studies with a longer follow-up period, i.e. a post-authorisation efficacy study (PAES) in 13 patients after 144 weeks of treatment, an extension study (190-202) of the initial study (190-201) in 23 patients with follow-up of around 5 years, a post-authorisation safety study (COMPASS), still ongoing, in 48 patients, including 8 French patients, with a mean (SD) treatment duration for all patients of 5.2 (2.05) years,
• comparative analyses, not without bias, for each of these studies on the efficacy of cerliponase alfa versus data from a historic cohort of patients not treated with cerliponase alfa, suggesting a benefit on the motricity/language score,
• safety data with a longer follow-up that did not evidence any new signals, the safety profile of BRINEURA (cerliponase alfa) being characterised by events associated with the medicinal product and its administration method by the intracerebroventricular route,
• the identified medical need in the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, a rare, genetic childhood disease rapidly progressing to disability and with a fatal outcome,

the Committee considers that BRINEURA 150 mg (cerliponase alfa) solution for infusion maintains a moderate clinical added value (CAV III) in the care pathway for neuronal ceroid lipofuscinosis type 2 (CLN2) disease.