Coverage Policies

Effective Date:

a)    This policy will apply to all services performed on or after the above revision date which will become the new effective date.

b)    For all services referred to in this policy that were performed before the revision date, contact customer service for the rules that would apply.

1)    Brineura (cerliponase) requires prior authorization.

2)    Brineura 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 know as tripeptidyl peptidase 1 (TPP1) deficiency.

3)    Brieneura is covered under the medical benefit.

I. Initial Approval Criteria

A. Late Infantile Neuronal Ceroid Lipofuscinosis Type 2 (must meet all):

1. Diagnosis of late infantile neuronal CLN2;

2. Prescribed by or in consultation with a neurologist;

3. Age ≥ 3 years;

4. Confirmation of CLN2 with both of the following (a and b):

a. TPP1 enzyme activity test demonstrating deficient TPP1 enzyme activity in leukocytes;

b. Identification of 2 pathogenic mutations in trans in the TPP1/CLN2 gene;

5. Motor domain of the CLN2 Clinical Rating Scale score ≥ 1;

6. At the time of request, member does not have ventriculoperitoneal shunts;

7. Dose does not exceed 300 mg administered once every other week as an intraventricular infusion.

Approval duration: 6 months

II. Continued Therapy

A. Late Infantile Neuronal Ceroid Lipofuscinosis Type 2 (must meet all):

1. Member is currently receiving medication via QualChoice benefit or member has previously met initial approval criteria;

2. Member is responding positively to therapy as evidenced a score of ≥ 1 on the CLN2 Clinical Rating Scale;

3. If request is for a dose increase, new dose does not exceed 300 mg administered once every other week as an intraventricular infusion.

Approval duration: 6 months

Codes Used In This BI:

1)    J0567 – Injection, cerliponase alfa, 1mg

1. Brineura Prescribing Information. Novato, CA: BioMarin Pharmaceutical Inc.; March 2020. Available at: https://www.brineura.com. Accessed May 2, 2022.

2. Williams RE, Adama HR, Blohm M, et al. Management strategies for CLN2 disease. Pediatric Neurology. 2017 Apr;(69):102-112. http://dx.doi.org/10.1016/j.pediatrneurol.2017.01.034.

3. Fietz M, AlSayed M, Burke D, et al. Diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2 disease): Expert recommendations for early detection and laboratory diagnosis. Molecular Genetics and Metabolism. 2016 Jul;(119):160-167. doi: 10.1016/j.ymgme.2016.07.011. Epub 2016 Jul 25.

4. Kohlschütter A, Schulz A, Bartsch U, et al. Current and Emerging Treatment Strategies for Neuronal Ceroid Lipofuscinoses. CNS Drugs (2019) 33:315-325. https://doi.org/10.1007/s40263-019-00620-8.