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DrugsBrineura™ (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

Renewal approval criteria

  • 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

Quantity limits

2 kits per 28 days


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

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