ModifiedAmeriHealth Caritas VIP CarePolicy MA08.078e

Sebelipase alfa (Kanuma)

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This policy governs medical-benefit coverage and prior authorization for intravenous sebelipase alfa (Kanuma) used to treat lysosomal acid lipase deficiency in pediatric and adult Medicare Advantage members. Coverage requires documented medical necessity, applicable dosing and frequency criteria, and compliance with the member's Evidence of Coverage; Part D pharmacy-benefit coverage is outside this policy.

Policy Summary

PayerAmeriHealth Caritas VIP Care

PolicySebelipase alfa (Kanuma)

Policy CodePolicy MA08.078e

Change TypeModified

Effective DateSeptember 26, 2022

Last ReviewSep 18, 2026

Next Review DateN/A

Key ActionSubmit prior approval for the requested dose and frequency, and submit any dose, frequency, or regimen change for a new approval.

SourceLink

POLICY UPDATE CHANGES

The September 26, 2022 version revised dosage and frequency requirements and definitions of suboptimal clinical response.

The September 26, 2022 version added dosage titration information for infants with rapidly progressive lysosomal acid lipase deficiency and for pediatric and adult individuals with lysosomal acid lipase deficiency.

The HCPCS code for sebelipase alfa was changed from C9478 to J2840 effective January 1, 2017.

The unclassified drugs or biologicals code C9399 was removed and replaced with C9478 effective July 1, 2016.

The policy was reviewed and reissued on September 18, 2026 to communicate the Company's continuing position on sebelipase alfa.

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ModifiedAmeriHealth Caritas VIP CarePolicy MA08.078e

Sebelipase alfa (Kanuma)

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Sign up for amerihealth_caritas_vip_care Policy MA08.078e alerts

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Policy Summary

PayerAmeriHealth Caritas VIP Care

PolicySebelipase alfa (Kanuma)

Policy CodePolicy MA08.078e

Change TypeModified

Effective DateSeptember 26, 2022

Last ReviewSep 18, 2026

Next Review DateN/A

Key ActionSubmit prior approval for the requested dose and frequency, and submit any dose, frequency, or regimen change for a new approval.

SourceLink

POLICY UPDATE CHANGES

The September 26, 2022 version revised dosage and frequency requirements and definitions of suboptimal clinical response.

The HCPCS code for sebelipase alfa was changed from C9478 to J2840 effective January 1, 2017.

The unclassified drugs or biologicals code C9399 was removed and replaced with C9478 effective July 1, 2016.

The policy was reviewed and reissued on September 18, 2026 to communicate the Company's continuing position on sebelipase alfa.

1 mg/kg IV weeklyInfant starting dose

1 mg/kg IV q2wkPediatric/adult starting dose

3 mg/kgEscalated dose

5 mg/kgMaximum infant dose

2 outcomesContinuation standard

Part BBenefit pathway

Coverage Criteria

Infants With Rapidly Progressive LAL Deficiency

Covered when ALL of the following criteria, including dosing and frequency requirements, are met:

ALL of the following

ONE of the following
- Confirmation of the absence or deficiency of lysosomal acid lipase (LAL) resulting from dried blood spot (DBS) testing.
- Confirmation of molecular genetic testing that reveals pathogenic variation(s) in the lipase A, lysosomal acid type (LIPA) gene, which results in reduction of functionality of LAL enzyme activity.
The infant presents within the first 6 months of life with documented clinical signs and symptoms of rapidly progressive LAL deficiency, such as diarrhea, vomiting, marked failure to thrive (e.g., weight decreasing across two or more major centiles on a standard World Health Organization [WHO] weight-for-age [WFA] chart; body weight below the 10th centile on a standard WHO WFA chart and no weight gain during the 2 weeks before screening; or loss of greater than 5 percent of birth weight after 2 weeks of age), abdominal distension, hepatomegaly, splenomegaly, rapidly progressive liver failure, adrenal calcifications, or anemia.
Dosing and frequency requirements
- Initial dosage is 1 mg/kg intravenously (IV) once weekly.
- If an optimal clinical response is not achieved, increase the dosage to 3 mg/kg once weekly.
- For continued suboptimal clinical response at 3 mg/kg once weekly, further increase the dosage to 5 mg/kg once weekly.
- A suboptimal clinical response within the first 3 months of treatment is present when ALL of the following apply
  - Required conditions
    Absence of other potential causes of any observed clinical manifestations.
    Absence of missed infusions.

Initial Therapy: Pediatric and Adult Individuals

Covered when ALL of the following criteria, including dosing and frequency requirements, are met:

ALL of the following

ONE of the following
- Confirmation of the absence or deficiency of lysosomal acid lipase (LAL) resulting from dried blood spot (DBS) testing.
- Confirmation of molecular genetic testing that reveals pathogenic variation(s) in the lipase A, lysosomal acid type (LIPA) gene, which results in reduction of functionality of LAL enzyme activity.
Documented presence of clinical signs and symptoms of LAL deficiency, such as hepatomegaly, dyslipidemia, increased serum transaminases (ALT, AST), evidence of advanced liver disease (cirrhosis, fibrosis, or steatosis), or histologically confirmed disease recurrence in individuals with a past liver or hematopoietic transplant more than 2 years prior.

Continuation Therapy

Covered for continuation therapy when the individual has improvement from baseline for the initial request for continuation therapy, or maintenance of previous improvements, as evidenced by at least TWO of the following:

ALL of the following

At least TWO of the following
- Improvement in ONE symptom or disease manifestation
  - For pediatric individuals, increase in growth and/or weight parameters based on expected anthropometric parameters.
  - Decrease in diarrhea and/or vomiting, measured by decreased frequency and/or volume.
  - Improvement in liver volume; normal liver volume in liters is defined as 2.5 percent of body weight in kilograms.
  - Improvement in liver fat content, defined as a decrease of greater than 5 percent in liver fat content from baseline as measured by magnetic resonance imaging (MRI); normal liver fat content is less than 5 percent.

When molecular genetic testing reveals established benign variation(s) or a wild-type genotype in the lipase A, lysosomal acid type (LIPA) gene, sebelipase alfa (Kanuma) is considered not medically necessary and, therefore, not covered because the available published peer-reviewed literature does not support its use in the treatment of this disease.

When molecular genetic testing reveals likely pathogenic variation(s) or variants of unknown significance (VUS) in the lipase A, lysosomal acid type (LIPA) gene, the use of sebelipase alfa (Kanuma) is considered experimental/investigational and, therefore, not covered because the safety and/or effectiveness of this service cannot be established by review of the available published peer-reviewed literature.

All other uses for sebelipase alfa (Kanuma) are considered experimental/investigational and, therefore, not covered unless the indication is supported as an accepted off-label use, as defined in the Company medical policy on off-label coverage for prescription drugs and biologics.

This policy only addresses instances when sebelipase alfa (Kanuma) is covered under a member's medical benefit (Part B benefit). It does not address instances when sebelipase alfa (Kanuma) is covered under a member's pharmacy benefit (Part D benefit).

When molecular genetic testing reveals established benign variation(s) or a wild-type genotype in the LIPA gene, sebelipase alfa (Kanuma) is not medically necessary and is not covered.

Initial Therapy Criteria

Infant Initial Therapy

For infants with rapidly progressive LAL deficiency presenting within the first 6 months of life, coverage requires all of the following.

Infant initial therapy

The infant presents within the first 6 months of life.
Confirmed LAL deficiency
- Dried blood spot testing confirms absence or deficiency of LAL.
- Molecular genetic testing reveals pathogenic LIPA variation(s) that reduce LAL enzyme functionality.
Clinical signs and symptoms are documented, including diarrhea, vomiting, marked failure to thrive, abdominal distension, hepatomegaly, splenomegaly, rapidly progressive liver failure, adrenal calcifications, or anemia.

Continuation Therapy Criteria

Coding and Clinical Thresholds

CPT Procedure Code Number(s)CPT

N/A

ICD-10 Procedure Code Number(s)ICD-10

N/A

ICD-10 Diagnosis Code Number(s)ICD-10Covered

E75.5

Other lipid storage disorders

HCPCS Level II Code Number(s)HCPCSCovered

J2840

Injection, sebelipase alfa, 1 mg

Revenue Code Number(s)Revenue

N/A

Historical HCPCS codesHCPCS

C9478	Injection, sebelipase alfa, 1 mg; historical code removed effective 01/01/2017
J3590	Unclassified biologics; historical code removed and replaced by J2840

Historical NOC codeHCPCS

C9399

Unclassified drugs or biologicals; historical code removed and replaced by C9478

Rapidly progressive infant presentation

PopulationInfants with rapidly progressive lysosomal acid lipase (LAL) deficiency

Age at presentationWithin the first 6 months of life

Coverage contextCoverage requires all applicable criteria, including dosing and frequency requirements

Poor growth response

Growth velocityFailure to gain an average of 5 g/kg body weight per day

Provider Actions and Documentation

Prior Authorization

Submit prior approval for dose and frequency

Prior approval is required for the requested dose and frequency under the utilization management process. If the member’s weight, therapeutic response, dose, frequency, or regimen changes, submit the change for a new approval.

Documentation Required

Support exceptions with literature

Requests for quantities outside the policy dosing and frequency requirements require supporting published peer-reviewed literature from the prescribing professional provider.

Documentation Required

Maintain medical necessity records

The medical record must reflect medical necessity for the care provided. Maintain applicable records, including professional provider, hospital, nursing home, home health, therapy, and test records, and make all documentation available to the Company upon request.

Clinical Background

Lysosomal acid lipase deficiency is an inherited disorder caused by deficient activity of lysosomal acid lipase, an enzyme that normally breaks down lipid substrates into free cholesterol and free fatty acids. Deficient activity results in progressive lysosomal accumulation of cholesteryl esters and triglycerides, with manifestations that may involve the liver, gastrointestinal system, growth, blood counts, and lipid profile. The rapidly progressive infant form, commonly associated with Wolman disease, may present in the first weeks of life with hepatosplenomegaly, gastrointestinal symptoms, malabsorption, growth failure, adrenal calcification, and liver failure. Pediatric and adult disease, also known as cholesteryl ester storage disease, generally has residual enzyme activity and may cause variable lipid abnormalities, hepatosplenomegaly, liver disease, and malabsorption. Sebelipase alfa is an intravenous enzyme replacement therapy for LAL deficiency.

Definitions

Suboptimal clinical response within first 3 months

TimingWithin the first 3 months of treatment

Required exclusionsNo alternative causes, missed infusions, acute cholecystitis, potentially hepatotoxic concomitant medication, or causative concomitant illness

Response findingsAt least two specified abnormal outcomes after at least 4 infusions at the current dose

Examples of abnormal outcomesPoor growth, albumin less than 3.5 g/dL, ALT more than twice ULN, ongoing blood or platelet transfusion requirement, or anti-drug, anti-enzyme, or anti-uptake antibodies

Suboptimal clinical response after first 3 months

TimingAfter the first 3 months of treatment

Site of Care

Note

Verify home-infusion benefit

Kanuma may be available under the medical benefit through home infusion providers. Verify the member’s home-infusion benefit before arranging this site of care.

Policy Summary

PayerAmeriHealth Caritas VIP Care

PolicySebelipase alfa (Kanuma)

Policy CodePolicy MA08.078e

Change TypeModified

Effective DateSeptember 26, 2022

Last ReviewSep 18, 2026

Next Review DateN/A

Key ActionSubmit prior approval for the requested dose and frequency, and submit any dose, frequency, or regimen change for a new approval.

SourceLink

On This Page

Absence of acute cholecystitis.

Absence of initiation of a potentially hepatotoxic concomitant medication.

Absence of a concomitant illness that could cause any of the observed lack of clinical responsiveness to the drug.

The infant has received at least 4 infusions at the current dose.

At least TWO of the following

Poor growth, defined as failure to gain an average of 5 g/kg body weight per day AND either a WHO weight-for-length or weight-for-height z score less than -2, or a WHO length-for-age or height-for-age z score less than -2.
Albumin less than 3.5 g/dL.
ALT more than twice the upper limit of normal (ULN).
Ongoing requirement for blood and/or platelet transfusion.
Presence of anti-drug, anti-enzyme, or anti-uptake antibodies.

A suboptimal clinical response after the first 3 months of treatment is present when ALL of the following apply

Required conditions
- Lack of improvement from a minimum of 3 assessments or failure to normalize within 12 months of treatment in the applicable clinical manifestations.
- Absence of other potential causes of any observed clinical manifestations.
- Absence of missed infusions.
- Absence of acute cholecystitis.
- Absence of initiation of a potentially hepatotoxic concomitant medication.
- Absence of a concomitant illness that could cause any of the observed lack of clinical responsiveness to the drug.
ONE or more of the following manifestations
- Decrease in weight-for-age crossing at least 2 major centiles.
- Albumin less than 3.5 g/dL.
- ALT more than twice the ULN.
- Persistent or worsening hepatomegaly, splenomegaly, or lymphadenopathy.
- Presence of anti-drug, anti-enzyme, or anti-uptake antibodies.

Improvement in liver histopathology, such as cirrhosis or fibrosis.

Improvement in ONE component of a lipid panel

Improvement in low-density lipoprotein cholesterol (LDL-c), based on clinical comparisons with baseline and/or previous results.
Improvement in high-density lipoprotein cholesterol (HDL-c), based on clinical comparisons with baseline and/or previous results.
Improvement in triglycerides (TG), based on clinical comparisons with baseline and/or previous results.

Improvement in ONE serum transaminase

Improvement in ALT, based on clinical comparisons with baseline and/or previous results.
Improvement in AST, based on clinical comparisons with baseline and/or previous results.

Pediatric and adult dosing and frequency requirements

Standard dosage is 1 mg/kg IV once every other week.
For a suboptimal clinical response or significant clinical progression, increase the dosage to 3 mg/kg once every other week.
Suboptimal clinical response after at least 8 consecutive infusions
- Absence of other potential causes of observed clinical manifestations, including initiation of a potentially hepatotoxic concomitant medication in an individual with abnormal AST or ALT, missed infusions, development of viral or autoimmune hepatitis, or development of another alternative etiology of liver disease.
- ONE of the following
  - ALT or AST remains abnormal and either has not improved from baseline or has worsened from the previously achieved lowest value over the preceding 8 consecutive infusions.
  - LDL-c or TG remains abnormal and either has not improved or has worsened over the preceding 8 consecutive infusions.
  - The individual is younger than 18 years at assessment and has a WFA z-score 2 standard deviations below the mean, either did not improve or worsened during the preceding 6 months, and did not miss more than 20 percent of infusions during the preceding 6 months.
Significant clinical progression after at least 5 consecutive infusions
- Absence of other potential causes of observed clinical manifestations, including initiation of a potentially hepatotoxic concomitant medication in an individual with abnormal AST or ALT, missed infusions, development of viral or autoimmune hepatitis, or development of another alternative etiology of liver disease.
- Confirmed elevation of ALT or AST to greater than 5 times the ULN and at least twice the highest pre-treatment value.
- ONE of the following
  - Increase of total bilirubin to greater than 3 times the ULN and at least twice the highest pre-treatment value.
  - Prolongation of prothrombin time (PT) 4 seconds or greater above baseline.
  - Development or worsening of ascites.

Additional serum laboratory studies that may be considered after the continuation criteria above have been reviewed

Improvement in gamma-glutamyl transferase (GGT), albumin, protein, total and conjugated bilirubin, alkaline phosphatase, lactate dehydrogenase, or hemoglobin, based on clinical comparisons with baseline and/or previous results.

Administer 1 mg/kg IV once weekly; increase to 3 mg/kg once weekly if optimal clinical response is not achieved, and to 5 mg/kg once weekly for continued suboptimal clinical response at 3 mg/kg.1, 3, or 5 mg/kg IV once weekly

Pediatric and Adult Initial Therapy

For pediatric and adult individuals with LAL deficiency, coverage requires all of the following.

Pediatric and adult initial therapy

Confirmed LAL deficiency
- Dried blood spot testing confirms absence or deficiency of LAL.
- Molecular genetic testing reveals pathogenic LIPA variation(s) that reduce LAL enzyme functionality.
Documented clinical manifestations
- Hepatomegaly is documented.
- Dyslipidemia is documented.
- Serum ALT or AST is increased.
- Advanced liver disease, including cirrhosis, fibrosis, or steatosis, is documented.
- Histologically confirmed disease recurrence is documented after a liver or hematopoietic transplant performed more than 2 years earlier.
Administer 1 mg/kg IV once every other week; increase to 3 mg/kg once every other week for suboptimal clinical response or significant clinical progression.1 or 3 mg/kg IV once every other week

Weight-for-length or weight-for-height

WHO z score less than -2

Length-for-age or height-for-ageWHO z score less than -2

Required findingPoor growth requires the growth-velocity finding together with either WHO z-score finding

Albumin

Laboratory testAlbumin

ThresholdLess than 3.5 g/dL

Clinical useOne of the specified findings used to assess suboptimal clinical response

ALT

Laboratory testALT

ThresholdMore than 2 times the upper limit of normal (ULN)

Clinical useOne of the specified findings used to assess suboptimal clinical response

Early response assessment

Assessment pointAfter receiving at least 4 infusions at the current dose

Assessment purposeEvaluate for suboptimal clinical response within the first 3 months of treatment

Response requirementTwo or more specified abnormal outcomes are required for the early suboptimal-response definition

Weight loss or failure to thrive

Weight trajectoryWeight decreasing across 2 or more major centiles on a standard WHO weight-for-age chart

Low weight with no gainBody weight below the 10th centile on a standard WHO weight-for-age chart and no weight gain during the preceding 2 weeks

Birth-weight lossLoss of greater than 5% of birth weight after 2 weeks of age

Liver fat improvement

Imaging methodMagnetic resonance imaging (MRI)

Improvement thresholdDecrease of greater than 5% in liver fat content from baseline

Normal liver fat contentLess than 5%

Normal liver volume

MeasurementLiver volume

Normal value2.5% of body weight in kilograms

Clinical useImprovement in liver volume is a qualifying continuation-therapy outcome

Pediatric growth response

AgeUnder 18 years of age at the time of assessment

Weight-for-age statusWFA z-score is 2 standard deviations below the mean

Recent growth trendNo improvement or worsening during the preceding 6 months

Infusion adherenceNo more than 20% of infusions missed during the preceding 6 months

Suboptimal response assessment

Assessment pointAfter at least 8 consecutive infusions

Alternative causesAbsence of other potential causes of observed clinical manifestations

Required findingAt least one qualifying persistent or worsening liver-test, lipid, or pediatric-growth finding

Significant progression liver enzyme threshold

Laboratory testsALT or AST

Absolute thresholdGreater than 5 times the upper limit of normal (ULN)

Pretreatment comparisonAt least twice the highest pretreatment value

Additional requirementThe enzyme elevation must occur with bilirubin elevation, PT prolongation, ascites, or encephalopathy

Significant progression bilirubin threshold

Laboratory testTotal bilirubin

Absolute thresholdGreater than 3 times the upper limit of normal (ULN)

Pretreatment comparisonAt least twice the highest pretreatment value

Clinical contextOne possible finding accompanying significant liver-enzyme progression

Significant progression PT threshold

Laboratory testProthrombin time (PT)

Threshold4 seconds or greater above baseline

Clinical contextOne possible finding accompanying significant liver-enzyme progression

Significant progression assessment

Assessment pointAfter at least 5 consecutive infusions

Alternative causesAbsence of other potential causes of observed clinical manifestations

Required liver findingALT or AST greater than 5 times ULN and at least twice the highest pretreatment value

Additional findingIncrease in total bilirubin, PT prolongation, development or worsening of ascites, or development of encephalopathy

Documentation Required

Document diagnostic confirmation and clinical findings

Submit confirmation of LAL deficiency through dried blood spot testing showing absence or deficiency of LAL, or molecular genetic testing showing pathogenic LIPA variation(s) that reduce LAL enzyme functionality. Also document the individual’s clinical signs and symptoms.

Documentation Required

Document response for continuation therapy

For continuation therapy, document improvement from baseline or maintenance of previous improvement in at least two qualifying outcomes. Qualifying outcomes include clinical symptoms or findings, a lipid-panel component, or a serum transaminase.

Documentation Required

Support out-of-guideline dosing

Requests outside the stated dosing and frequency requirements must include published peer-reviewed literature supporting the requested amount or regimen.

Denial Risk

Benign or wild-type LIPA result

Sebelipase alfa is not medically necessary and is not covered when molecular genetic testing reveals established benign variation(s) or a wild-type genotype in the LIPA gene.

Denial Risk

Unapproved or unsupported use

All other uses are considered experimental/investigational and are not covered unless the indication is supported as an accepted off-label use under the Company’s policy for off-label coverage for prescription drugs and biologics.

Denial Risk

Likely pathogenic or VUS result

Use is considered experimental/investigational and is not covered when molecular genetic testing reveals likely pathogenic variation(s) or a variant of unknown significance in the LIPA gene.

Denial Risk

Produce requested records

Failure to produce requested medical records or other supporting documentation may result in denial of the drug. All documentation must be available to the Company upon request.

Billing Rule

Obtain approval before dose or regimen changes

A changed dose, frequency, or regimen requires a new approval based on the change. Claims for sebelipase alfa may also be subject to postpayment review and audit procedures.

Required exclusionsNo alternative causes, missed infusions, acute cholecystitis, potentially hepatotoxic concomitant medication, or causative concomitant illness

Response assessmentLack of improvement from a minimum of 3 assessments or failure to normalize within 12 months of treatment

Required clinical findingAt least one specified ongoing manifestation, such as weight-for-age decline across at least 2 major centiles, low albumin, elevated ALT, persistent or worsening organ enlargement, or relevant antibodies

Suboptimal clinical response

PopulationPediatric and adult individuals with LAL deficiency

Assessment timingAfter at least 8 consecutive infusions

Required exclusionAbsence of other potential causes, including hepatotoxic medication, missed infusions, viral or autoimmune hepatitis, or another alternative liver-disease etiology

Qualifying findingsPersistent or worsening abnormal ALT or AST, LDL-c or triglycerides, or qualifying pediatric growth findings

Significant clinical progression

Assessment timingAfter at least 5 consecutive infusions

Required exclusionAbsence of other potential causes of observed clinical manifestations

Liver enzyme criterionALT or AST greater than 5 times ULN and at least twice the highest pretreatment value

Additional laboratory or clinical criterionTotal bilirubin greater than 3 times ULN and at least twice the highest pretreatment value, PT 4 seconds or greater above baseline, ascites, or encephalopathy

Wolman disease

Disease formRapidly progressive form of lysosomal acid lipase deficiency

Typical presentationWithin the first weeks of life

Common manifestationsProminent hepatosplenomegaly, diarrhea and vomiting, malabsorption, growth failure, adrenal gland calcification, and liver failure

Disease courseAffected infants usually die within six to twelve months

Cholesteryl ester storage disease (CESD)

Alternative nameCholesteryl ester storage disease (CESD)

PopulationPediatric and adult individuals with LAL deficiency

Enzyme activityGenerally associated with some residual enzyme activity

Typical manifestationsSerum lipid abnormalities, hepatosplenomegaly, and/or elevated liver enzymes

Disease progressionVariable, with morbidity and mortality related to atherosclerosis, liver disease or liver failure, complications of hypersplenism, and/or malabsorption

Lysosomal acid lipase (LAL)

EnzymeLysosomal acid lipase (LAL)

Primary functionBreaks down lipid into free cholesterol and free fatty acids

Deficiency effectCauses progressive lysosomal accumulation of cholesteryl esters and triglycerides

Affected organs and tissuesIncludes the liver, spleen, intestine, and walls of the blood vessels

Development of encephalopathy.