Pharmacy Prior Authorization Criteria

Brineura (cerliponase alfa)

Indications for Prior Authorization

Brineura (cerliponase alfa)

• For diagnosis of Neuronal Ceroid Lipofuscinosis Type 2
  Indicated to slow the loss of ambulation in pediatric patients with neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency.

Criteria

Brineura

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)

• Diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2) (also known as tripeptidyl peptidase 1 (TPP1) deficiency)
AND
• Diagnosis is confirmed by tripeptidyl peptidase 1 (TPP1) enzyme detected by a dried blood spot test and CLN2 genotype analysis
AND
• Patient does not have acute intraventricular access-related complications (e.g., leakage, device failure, or device-related infections)
AND
• Patient does not have ventriculoperitoneal shunts
AND
• Prescribed by or in consultation with a neurologist with expertise in the diagnosis of CLN2
AND
• Administered in a healthcare setting by, or under the direction of, a physician knowledgeable in intraventricular administration and hypersensitivity reactions including anaphylaxis [A]

Brineura

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)

• Patient does not have acute intraventricular access-related complications (e.g., leakage, device failure, or device-related infections)
AND
• Patient does not have ventriculoperitoneal shunts
AND
• Administered in a healthcare setting by, or under the direction of, a physician knowledgeable in intraventricular administration and hypersensitivity reactions including anaphylaxis [A]
AND
• Patient demonstrates positive clinical response to therapy (e.g., improvement in walking or crawling, or no evidence of disease progression)

P & T Revisions

2024-09-04, 2024-05-01, 2023-06-26, 2023-06-19, 2022-04-25, 2021-04-22, 2020-04-09

References

1. Batten Disease Support and Research Association: Batten Disease Neuronal Ceroid Lipofuscinosis. Available at: http://bdsra.org/wp-content/uploads/2012/01/Batten-Disease-An-Easy-To-Understand-Guide.pdf. Accessed March 29, 2022.
2. Brineura Prescribing Information. BioMarin Pharmaceutical Inc. Novato, CA. July 2024.
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 Sep;119(1-2):160-7.
4. National Institutes of Health (NIH). Bethesda, MD. CLN2 Disease. Available at: https://ghr.nlm.nih.gov/condition/cln2-disease. Accessed March 29, 2022.

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End Notes

1. Brineura (cerliponase alfa) is for intraventricular use only and should be administered by, or under the direction of a physician knowledgeable in intraventricular administration. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Administration of Brineura should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. Initiate Brineura in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. [2]

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Revision History

• 2024-09-04: Criteria updated due to expanded indication.
• 2024-05-01: 2024 Annual Review. No criteria updates.
• 2023-06-26: Update effective date
• 2023-06-19: Annual Review
• 2022-04-25: Annual Review
• 2021-04-22: Annual Review
• 2020-04-09: Annual Review

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