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Brineura (cerliponase alfa)

Policy Number: PH-0299

 

(Intraventricular) 

Last Review Date: 02/04/2019

Date of Origin: 5/30/2017

Dates Reviewed: 05/2017, 04/2018, 02/2019

 

  1. Length of Authorization

           Coverage will be provided for 6 months and may be renewed.

  1. Dosing Limits
  1. Quantity Limit (max daily dose) [Pharmacy Benefit]:
  • Brineura 150 mg/5 mL single dose vial : 2 vials every 14 days
  1. Max Units (per dose and over time) [Medical Benefit]:
  • 300 billable units (two kits) every 14 days
  1. Initial Approval Criteria

Coverage is provided in the following conditions:

  • Patient is 3 years of age or older; AND
  • Patient must not have acute intraventricular access device-related complications (e.g., leakage, device failure, or device-related infection); AND
  • Patient must not have ventriculoperitoneal shunts; AND
  • Patient has no sign or symptom of acute, unresolved localized infection on or around the device insertion site (e.g. cellulitis or abscess); or a suspected or confirmed CNS infection; AND

Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2); tripeptidyl peptidase 1 (TPP1) deficiency †

  • Patient must have a definitive diagnosis of late infantile CLN2 confirmed by deficiency of the lysosomal enzyme tripeptidyl peptidase-1 (TPP1) and/or molecular analysis indicating dysfunctional mutation of the TPP1 gene on chromosome 11p15; AND
  • Patient has mild to moderate disease documented by a two-domain score of 3- 6 on motor and language domains of the Hamburg CLN2 Clinical Rating Scale, with a score of at least 1 in each of these two domains; AND
  • Patient is ambulatory; AND
  • Patients with a history of bradycardia, conduction disorder, or with structural heart disease must have electrocardiogram (ECG) monitoring performed during the infusion

FDA-labeled indication(s)

  1. Renewal Criteria
  • Patient continues to meet the criteria in section III; AND
  • Absence of unacceptable toxicity from the drug or complications from the device. Examples include the following: meningitis and other intraventricular access device-related infections, intraventricular access device-related complications, severe hypersensitivity reaction, severe cardiovascular reactions, severe hypotension; etc.; AND
  • Patient had a 12-lead ECG evaluation performed within the last 6 months (those with cardiac abnormalities require ECG during each infusion); AND
  • Patient has responded to therapy compared to pretreatment baseline with stability/lack of decline in motor function/milestones on the Motor domain of the Hamburg CLN2 Clinical Rating Scale (decline is defined as having an unreversed (sustained) 2-category decline or an unreversed score of 0)
  1. Dosage/Administration

Indication

Dose

CLN2

300 mg administered once every other week by intraventricular infusion. Administer Brineura first followed by infusion of the Intraventricular Electrolytes each at an infusion rate of 2.5 mL/hr. The complete Brineura infusion, including the required infusion of Intraventricular Electrolytes, is approximately 4.5 hours.

  • Aseptic technique must be strictly observed during preparation and administration
  • Brineura should be administered by, or under the direction of a physician knowledgeable in intraventricular administration
  • Brineura is administered into the cerebrospinal fluid (CSF) by infusion via a surgically implanted reservoir and catheter (intraventricular access device). Brineura is intended to be administered via the Codman® HOLTER RICKHAM Reservoirs with the Codman® Ventricular Catheter. The intraventricular access device must be implanted prior to the first infusion. It is recommended that the first dose be administered at least 5 to 7 days after device implantation
  • Brineura is intended to be administered with the B Braun Perfusor® Space Infusion Pump System
  • Pre-treatment of patients with antihistamines with or without antipyretics or corticosteroids is recommended 30 to 60 minutes prior to the start of infusion.

Store upright in freezer (-25C to -15C); thaw at room temperature for ~60 minutes prior to administration

  1. Billing Code/Availability Information

Jcode:

  • J0567 - Injection, cerliponase alfa, 1 mg: 1 billable unit = 1 mg (effective 1/1/19)

NDC:

  • Brineura 150 mg/5 mL (30 mg/mL) solution, two single-dose vials per carton co-packaged with Intraventricular Electrolytes Injection 5 mL in a single-dose vial: 68135-0811-xx
  1. References
  1. Brineura [package insert]. Novato, CA; BioMarin Pharmaceutical Inc.; December 2018. Accessed January 2019.
  2. Schulz A, Specchio N, Gissen P. Intracerebroventricular cerliponase alfa (BMN 190) in children with CLN2 disease: Results from a Phase 1/2, open-label, dose-escalation study. J Inherit Metab Dis. 2016;39(Suppl. 1):S51.
  3. Cherukuri A, Cahan H, Van Tuyl A, et al. Immunogenicity to cerliponase alfa, an enzyme replacement therapy for patients with CLN2 disease: results from a phase 1/2 study. Molecular Genetics and Metabolism. 2017 Jan 1;120(1):S35.
  4. Schulz A, Specchio N, Gissen P, et al. Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: interim results from an ongoing multicenter, multinational extension study. Molecular Genetics and Metabolism. 2017 Jan 1;120(1):S120.
  5. Mole SE, Williams RE. Neuronal Ceroid-Lipofuscinoses. GeneReviews. www.ncbi.nlm.nih.gov/books/NBK1428/ (Accessed on May 01, 2017).
  6. Online Mendelian Inheritance in Man, OMIM®. Johns Hopkins University, Baltimore, MD. MIM Number: 204500: 9/18/201 World Wide Web URL: https://omim.org/

Appendix 1 – Covered Diagnosis Codes

ICD-10

ICD-10 Description

E75.4

Neuronal ceroid lipofuscinosis

Appendix 2 – Centers for Medicare and Medicaid Services (CMS)

Medicare coverage for outpatient (Part B) drugs is outlined in the Medicare Benefit Policy Manual (Pub. 100-2), Chapter 15, §50 Drugs and Biologicals. In addition, National Coverage Determination (NCD) and Local Coverage Determinations (LCDs) may exist and compliance with these policies is required where applicable. They can be found at: http://www.cms.gov/medicare-coverage-database/search/advanced-search.aspx. Additional indications may be covered at the discretion of the health plan.

Medicare Part B Covered Diagnosis Codes (applicable to existing NCD/LCD):N/A

Medicare Part B Administrative Contractor (MAC) Jurisdictions

Jurisdiction

Applicable State/US Territory

Contractor

E (1)

CA, HI, NV, AS, GU, CNMI

Noridian Healthcare Solutions, LLC

F (2 & 3)

AK, WA, OR, ID, ND, SD, MT, WY, UT, AZ

Noridian Healthcare Solutions, LLC

5

KS, NE, IA, MO

Wisconsin Physicians Service Insurance Corp (WPS)

6

MN, WI, IL

National Government Services, Inc. (NGS)

H (4 & 7)

LA, AR, MS, TX, OK, CO, NM

Novitas Solutions, Inc.

8

MI, IN

Wisconsin Physicians Service Insurance Corp (WPS)

N (9)

FL, PR, VI

First Coast Service Options, Inc.

J (10)

TN, GA, AL

Palmetto GBA, LLC

M (11)

NC, SC, WV, VA (excluding below)

Palmetto GBA, LLC

L (12)

DE, MD, PA, NJ, DC (includes Arlington & Fairfax counties and the city of Alexandria in VA)

Novitas Solutions, Inc.

K (13 & 14)

NY, CT, MA, RI, VT, ME, NH

National Government Services, Inc. (NGS)

15

KY, OH

CGS Administrators, LLC