Summary

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• Brineura (cerliponase alfa) is indicated to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency.
• This summary is based on the review of one randomized controlled trial(s). ^[1]
• The median time until a 2-point decline in motor-language score was not reached for patients treated with cerliponase alfa, whereas historical controls reached this decline in a median of 345 days.
• Treated patients exhibited a mean unadjusted rate of decline in motor-language score of 0.27 ± 0.35 points per 48-week period, compared to 2.12 ± 0.98 points per 48-week period in historical controls, with a mean difference of 1.85 points (P<0.001).
• The study focused on children aged 3 to 16 years with CLN2 disease, with no specific differences in effectiveness outcomes among subgroups within this population mentioned.
• Common adverse events in children aged 3 to 16 years with CLN2 disease treated with cerliponase alfa included convulsions, pyrexia, vomiting, hypersensitivity reactions, and failure of the intraventricular device.
• Serious adverse events involved device-related infections in 2 patients, which necessitated antibiotic treatment and device replacement.
• There is no population types or subgroups information available in the reviewed studies.