Pharmacy Prior Authorization Criteria

Kanuma (sebelipase alfa)

For diagnosis of Lysosomal Acid Lipase (LAL) deficiency
Indicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency.

Prior Authorization (Initial Authorization)

Length of Approval: 12 Month(s)

Diagnosis of lysosomal acid lipase deficiency (LAL-D, Wolman Disease, Cholesteryl ester storage disease) [B]

AND

Enzymatic blood test (e.g., dried blood spot test) demonstrating a deficiency of LAL enzyme activity

Genetic testing for mutations in the lipase A, lysosomal acid type (LIPA) gene

AND

Prior Authorization (Reauthorization)

Length of Approval: 24 Month(s)

Patient demonstrates positive clinical response to therapy (e.g., reduction in LDL, triglycerides, AST or ALT, increase in HDL, reduction in liver fat content)

AND

2024-07-09, 2023-12-20, 2023-11-01, 2023-10-10, 2023-07-03, 2022-07-01, 2021-07-07, 2021-05-19, 2020-06-16

Kanuma prescribing information, Alexion Pharmaceuticals. Cheshire, CT. November 2021.
Burton BK, Balwani M, Feillet F, et al. A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency. N Engl J Med. 2015;373(11):1010-20.
Reiner, Guardamagna, Nair, et al. Lysosomal acid lipase deficiency - an under-recognized cause of dyslipidaemia and liver dysfunction. Atherosclerosis. 2014;235(1): 21-30.
Strebinger G, Müller E, Feldman A, Aigner E. Lysosomal acid lipase deficiency - early diagnosis is the key. Hepat Med. 2019 May 23;11:79-88.

Due to similar clinical presentations, LAL-D is often misdiagnosed as familial defective apolipoprotein B (ApoB) deficiency, heterozygous familial hypercholesterolemia (HeFH), familial combined hyperlipidemia (FCH), or polygenic hypercholesterolaemia [3]. A diagnosis of LAL-D can be confirmed by identification of a LIPA mutation or a deficient LAL enzyme in peripheral blood leukocytes, fibroblasts, or dried blood spots. A biopsy and/or radiographic findings may help support a LAL-D diagnosis, however these are not considered diagnostic. [2,3]
LAL deficiency is sub-classified as Wolman disease in infants and cholesteryl ester storage disease (CESD) in children and adults. [4]

2024-07-09: Annual review: No criteria changes.
2023-12-20: Program update to standard reauthorization language. No changes to clinical intent.
2023-11-01: Program update to standard reauthorization language. No changes to clinical intent.
2023-10-10: Patient demonstrates positive clinical response to therapy
2023-07-03: Annual review: No criteria changes. Updated initial auth duration to 12 months and reauth duration to 24 months.
2022-07-01: Annual Review - criteria updated to have initial and reauth criteria and respective approval lengths updated; specialist requirement options expanded
2021-07-07: Annual Review
2021-05-19: Addition of EHB formulary to guideline, no changes to criteria
2020-06-16: Annual Review - No Changes