Coverage is subject to the specific terms of the member's benefit plan.
The use of cerliponase alfa (Brineura) may be considered medically necessary when ALL of the following criteria are met:
- The individual must be between 3 and 8 years of age; and
- The individual must have diagnosis of late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency confirmed by the following:
- Molecular analysis that has detected two pathogenic variants/mutations in the TPP1/CLN2 gene; and
- An enzyme assay confirming deficiency of tripeptidyl peptidase 1 (TPP1); and
- Brineura must be prescribed by or in consultation with a metabolic specialist, geneticist, or pediatric neurologist; and
- The individual must not have ventriculoperitoneal shunts; and
- Baseline results of motor and language domains of the Hamburg CLN2 Clinical Rating Scale must be submitted and meet the following parameters
- Results must show a combined score of less than 6 in the motor and language domains; and
- Results must show a score of at least 1 in each of these domains.
Initial Authorization: 6 months
Continuation of therapy with cerliponase alfa (Brineura) may be considered medically necessary when ALL of the following are met:
- The individual must not have acute, unresolved localized infection on or around the device insertion site or suspected or confirmed CNS infection; and
- Individual maintains at a score of at least 1 in the motor domain on the Hamburg CLN2 Clinical Rating Scale; and
- The individual has responded to therapy compared to pretreatment baseline with stability/lack of decline* in motor function/milestones.
*: Decline is defined as having an unreversed (sustained) 2-category decline or an unreversed score of 0 in the Motor domain of the CLN2 Clinical Rating Scale
Continuation Authorization: 12 months
Cerliponase alfa (Brineura) is considered experimental/investigational for any other indication and therefore non-covered. Scientific evidence does not support its use for any other indications.