cerliponase alfa (Brineura)

^[1]^[2]^[3]

Indications

symptomatic pediatric patients >= 3 years of age with
late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), a form of Batten disease

* improves motor & language function^[2]

300 mg administered once every other week as an intraventricular infusion (2.5 mL/hr) followed by infusion of intraventricular electrolytes
duration of total infusion over ~4.5 hours

single-dose vials 5 mL, 2/carton: 30 mg/mL

co-packaged with intraventricular electrolytes solution 5 mL in a single dose vial

↑ Windle ML. FDA New Drug and Biologic Approvals -- 2017 Year-in-Review Medscape - Jan 11, 2018. https://reference.medscape.com/viewarticle/890871
U.S. Food and Drug Administration. Novel Drug Approvals for 2017. https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm537040.htm
↑ ^2.0 ^2.1 ^2.2 Schulz A, Ajayi T, Specchio N et al Study of Intraventricular Cerliponase Alfa for CLN2 Disease. N Engl J Med 2018; 378:1898-1907. May 17, 2018 PMID: https://www.ncbi.nlm.nih.gov/pubmed/29688815 Free full text https://www.nejm.org/doi/full/10.1056/NEJMoa1712649
↑ Brineura prescribing information http://brineura.com/downloads/Brineura_PI.pdf
Brineura (cerliponase alfa) prescribing information. BioMarin Pharmaceuticals, Inc. Novato, CA. April 2017. https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/761052lbl.pdf