FDA New Drug and Biologic Approvals -- 2017 Year-in-Review

Mary L Windle, PharmD

Disclosures

January 11, 2018

In This Article

Brineura (cerliponase alfa)

Indication:

Neuronal ceroid lipofuscinosis type 2 (CLN2, also known as tripeptidyl peptidase 1 [TPP1]) deficiency in children aged 3 years or older.

Mechanism:

Recombinant form of human tripeptidyl peptidase (TPP1) that provides enzyme replacement and restores breakdown of the lysosomal storage materials that cause CLN2 disease.

Dosage:

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:

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.

Reference:

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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