Product Details

Kanuma

Sebelipase alfa
2 mg/mL
Concentrate for solution for infusion
Single-Use 10-mL Vial Pack (Preservative-Free)

DIN/PIN/NPN

02469596

Manufacturer

Alexion Pharma GmbH

Formulary Listing Date

2021-04-08  

Unit Price

8546.0000

Amount MOH Pays

8546.0000

Coverage Status

Exceptional Access Program Product

ODB Formulary Therapeutic Classification

Therapeutic Note

NO

ATC Code

A16AB14

Interchangeable Products

NO  

LU Clinical Criteria

NO  

EAP Criteria

Therapeutic Class Reimbursement Criteria
Metabolic/Genetic Modifiers

Sebelipase alfa

  • Brand(s): Kanuma
  • Dosage Form/Strength: 2 mg/mL solution
  • Effective date: April 8, 2021

Initiation Criteria: 

For the treatment of lysosomal acid lipase (LAL) deficiency in patients meeting ALL the following criteria:

  1. Has documented biochemical evidence of deficient LAL activity; AND 

  2. Has two documented pathogenic mutations in the LIPA gene; AND

  3. Is under the care of a specialist with experience in the diagnosis and management of LAL deficiency; AND

  4. The onset of clinical manifestations of LAL deficiency occurred before six months of age;

    OR

  5. Patient is six months of age or older; AND
    i) Presents with one or more of the following:
    •     Persistently elevated transaminases (Alanine aminotransferase (ALT) > 1.5 x ULN1 or Aspartate transaminase (AST) > 1.5 x ULN1) as measured by two assessments three to six months apart
    •     Persistent dyslipidemia (Low-density lipoprotein cholesterol (LDL-C) and/or Triglycerides (TG) values in the top 5th percentile based on sex and age) as measured by two assessments three to six months apart
    •     Any documented hepatomegaly or hepatosplenomegaly
    •     Liver fibrosis confirmed by biopsy
    •     Failure to thrive
    •     Growth impairment2
    •     Evidence of intestinal affection and/or malabsorption;

    AND
    ii) Does not present with any of the following:
    •     Increased portal vein pressures, or de novo evidence of portal hypertension on ultrasound and Doppler, or new clinical presentation of portal hypertension (e.g., esophageal varices);
    OR
    •     Severe hepatic dysfunction (Child-Pugh Class C);
    OR
    •     End-stage liver disease.

NOTE: The requesting prescriber must provide baseline values for the following clinical components before starting sebelipase alfa; Bloodwork for ALT, AST, liver fibrosis, growth curve, spleen and liver volume/sizes, portal vein pressures, patient’s age, weight, and height. 

Discontinuation Criteria: 

Reimbursement will be discontinued in patients who have experienced adverse events from sebelipase alfa (particularly hypersensitivity reactions, including anaphylaxis, hypotension, or fever), which cannot be managed with standard treatment, and/or which have a significant impact on the patient’s quality of life, or are life-threatening.

For patients with onset of clinical manifestations of LAL deficiency at six months of age and older, reimbursement will be discontinued if: 

  • Patient has progressed to end-stage liver failure or multi-organ failure; OR 

  • Patient has at least three out of the five following response components compared with baseline values after 12 months of therapy: 

  1. Less than 10% improvement in ALT or AST 

  1. Worsening of liver fibrosis confirmed by biopsy 

  1. Persisting growth impairment2 despite sebelipase alfa therapy and nutritional interventions 

  1. At least a 15% increase in spleen volume and/or greater than 15% increase in liver volume on ultrasound 

  1. Increased portal vein pressures, or de novo evidence of portal hypertension on ultrasound and Doppler, or new presentation of portal hypertension (e.g., esophageal varices) 

1Based on age- and sex-specific normal values for ALT and AST 

2Growth impairment is defined as decreased body weight across at least two of the major centiles on a World Health Organization (WHO) weight-for-age chart, or body weight below 10th centile and no weight gain within two weeks and/or decreased height across at least two of the major centiles on a WHO height-for-age chart
(URLs: https://www.who.int/toolkits/child-growth- standards/standards/weight-for-age, https://www.who.int/toolkits/child-growth- standards/standards/length-height-for-age) 

Exclusion Criteria: Patients with severe liver disease and/or those who have progressed to end stage liver disease. 

Renewal Criteria: Renewals will be considered in patients who do not meet the discontinuation criteria. 

Recommended dose: 

- For patients with onset of clinical manifestations of LAL deficiency before six months of age:
1mg/kg administered as an intravenous infusion once weekly. Based on clinical response, dose increase to 3mg/kg may be considered. 

- For patients with onset of clinical manifestations of LAL deficiency at six months of age or older:
1mg/kg administered as an intravenous infusion once every other week. 

Approval duration of initials and renewals: 12 months initial, 6 months renewal

EAP Drug Request Form:

Standard Form for EAP Drug Requests

Product Monograph

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