Brineura™

Drugs - Brineura™ (cerliponase alfa) [Biomarin]

October 2017

Therapeutic area - tripeptidyl peptidase 1 (TPP1) deficiency

Initial approval criteria

Patient is 3 years of age or older AND
Patient must have a definitive diagnosis of late infantile CLN2 confirmed by deficiency of the lysosomal enzyme tripeptidyl peptidase-1 (TPP1) AND
Patient has symptomatic disease (e.g., seizures, motor decline, cognitive decline, decreased visual acuity, etc.) AND
Patient is ambulatory AND
Patient must not have ventriculoperitoneal shunts AND
Patient must not have acute intraventricular access device-related complications (e.g., leakage, device failure, or device-related infection) AND
Patients with a history of bradycardia, conduction disorder, or with structural heart disease must have electrocardiogram (ECG) monitoring performed during the infusion AND
Baseline documentation of pretreatment motor function/milestones, including but not limited to, the following validated scale: the Motor domain of a Hamburg CLN2 Clinical Rating Scale, etc.
Initial approval is for 6 months

Patient continues to meet the criteria above AND
Absence of unacceptable toxicity from the drug or complications from the device. Examples include the following: intraventricular access device leakage or infection, severe hypersensitivity reaction, 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 validated scale such as the Motor domain of the CLN2 Clinical Rating Scale, etc.
- Note: 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
Renewal approval is for 12 months

2 kits per 28 days

MHCP Provider Call Center 651-431-2700 or 800-366-5411