Neuronal ceroid lipofuscinosis type 2 (CLN2, also known as tripeptidyl peptidase 1 [TPP1]) deficiency in children aged 3 years or older.
Recombinant form of human tripeptidyl peptidase (TPP1) that provides enzyme replacement and restores breakdown of the lysosomal storage materials that cause CLN2 disease.
Administered to the cerebrospinal fluid by infusion via a surgically implanted reservoir and catheter.
300 mg by intraventricular infusion at rate of 2.5 mL/hr once every other week. Follow with intraventricular infusion of electrolytes at rate of 2.5 mL/hr. The total infusion time is about 4.5 hours.
See prescribing information for complete administration details.
Approval was based on a nonrandomized, single-arm dose escalation study over 96 weeks. Results were compared with untreated patients from a natural history cohort. Twenty-four patients aged 3-8 years were enrolled in the clinical study. One patient withdrew after week 1 due to inability to continue with study procedures; 23 patients were treated with cerliponase alfa every other week for 48 weeks and continued treatment during the extension. Twenty-two patients were evaluated at week 96, and 21 (95%) did not have a decline in the motor domain of the CLN2 clinical rating scale. Only the patient who terminated early was deemed to have a decline in the motor domain of the CLN2 clinical rating scale.
Brineura (cerliponase alfa) prescribing information. BioMarin Pharmaceuticals, Inc. Novato, CA. April 2017. https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/761052lbl.pdf
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Cite this: Mary L Windle. FDA New Drug and Biologic Approvals -- 2017 Year-in-Review - Medscape - Jan 11, 2018.