Notification

Alglucosidase alfa (e.g., Lumizyme®)


Notification Issue Date: 03/05/2019

This version of the policy will become effective 06/03/2019.

This policy has been updated to communicate dosing and frequency requirements for alglucosidase alfa (e.g., Lumizyme®) and requirements for laboratory and/or genetic testing consistent with the US Food and Drug Administration (FDA) labeling.



Medical Policy Bulletin


Title:Alglucosidase alfa (e.g., Lumizyme®)

Policy #:08.00.72h

This policy is applicable to the Company’s commercial products only. Policies that are applicable to the Company’s Medicare Advantage products are accessible via a separate Medicare Advantage policy database.


The Company makes decisions on coverage based on Policy Bulletins, benefit plan documents, and the member’s medical history and condition. Benefits may vary based on contract, and individual member benefits must be verified. The Company determines medical necessity only if the benefit exists and no contract exclusions are applicable.

When services can be administered in various settings, the Company reserves the right to reimburse only those services that are furnished in the most appropriate and cost-effective setting that is appropriate to the member’s medical needs and condition. This decision is based on the member’s current medical condition and any required monitoring or additional services that may coincide with the delivery of this service.

This Medical Policy Bulletin document describes the status of medical technology at the time the document was developed. Since that time, new technology may have emerged or new medical literature may have been published. This Medical Policy Bulletin will be reviewed regularly and be updated as scientific and medical literature becomes available. For more information on how Medical Policy Bulletins are developed, go to the About This Site section of this Medical Policy Web site.



Policy

Coverage is subject to the terms, conditions, and limitations of the member's contract.

MEDICALLY NECESSARY

Alglucosidase alfa (Lumizyme®) is considered medically necessary and, therefore, covered for the treatment of symptomatic individuals one month of age and older with infantile-onset or juvenile/adult-onset Pompe disease (GAA deficiency) when all of the following criteria are met, including dosing and frequency:
  • Diagnosis is confirmed by either of the following:
    • Deficiency of acid alpha-glucosidase in leukocytes or skin fibroblasts or muscles
    • Confirmation of biallelic pathogenic variant(s) in the GAA gene.
  • Dosing and frequency: infants one month of age and older, children, adolescents and adult dosing IV: 20 mg/kg every 2 weeks.

NOT ELIGIBLE FOR REIMBURSEMENT

Alglucosidase alfa (Myozyme®) is no longer manufactured and has been withdrawn from market; therefore, it is not eligible for reimbursement.

EXPERIMENTAL/INVESTIGATIONAL

All other uses of alglucosidase alfa (Lumizyme®) 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.

DOSING AND FREQUENCY REQUIREMENTS

The Company reserves the right to modify the Dosing and Frequency Requirements listed in this policy to ensure consistency with the most recently published recommendations for the use of alglucosidase alfa (Lumizyme®). Changes to these guidelines are based on a consensus of information obtained from resources such as, but not limited to: the US Food and Drug Administration (FDA); Company-recognized authoritative pharmacology compendia; or published peer-reviewed clinical research. The professional provider must supply supporting documentation (i.e., published peer-reviewed literature) in order to request coverage for an amount of alglucosidase alfa (Lumizyme®) outside of the Dosing and Frequency Requirements listed in this policy. For a list of Company-recognized pharmacology compendia, view our policy on off-label coverage for prescription drugs and biologics.

Accurate member information is necessary for the Company to approve the requested dose and frequency of this drug. If the member’s dose, frequency, or regimen changes (based on factors such as changes in member weight or incomplete therapeutic response), the provider must submit those changes to the Company for a new approval based on those changes as part of the utilization management activities. The Company reserves the right to conduct post-payment review and audit procedures for any claims submitted alglucosidase alfa (Lumizyme®).

REQUIRED DOCUMENTATION

The individual's medical record must reflect the medical necessity for the care provided. These medical records may include, but are not limited to: records from the professional provider's office, hospital, nursing home, home health agencies, therapies, and test reports.

The Company may conduct reviews and audits of services to our members, regardless of the participation status of the provider. All documentation is to be available to the Company upon request. Failure to produce the requested information may result in a denial for the drug.

When coverage of alglucosidase alfa (Lumizyme®) is requested outside of the Dosing and Frequency Requirements listed in this policy, the prescribing professional provider must supply documentation (i.e., published peer-reviewed literature) to the company that supports this request.
Guidelines

Per the US Food and Drug Administration (FDA)--approved labeling, the recommended dosing and frequency of alglucosidase alfa (Lumizyme®) is 20mg/kg body weight administered by intravenous (IV) infusion every two weeks.

BLACK BOX WARNING

Refer to the specific manufacturer's prescribing information for any applicable Black Box Warnings.

BENEFIT APPLICATION

Subject to the terms and conditions of the applicable benefit contract, alglucosidase alfa (Lumizyme®) is covered under the medical benefits of the Company’s products when the medical necessity criteria including dosing and frequency requirements listed in this medical policy are met.

US FOOD AND DRUG ADMINISTRATION (FDA) STATUS

Under a priority review, the FDA approved alglucosidase alfa (Lumizyme®) as an orphan drug on May 10, 2010.

PEDIATRIC USE

The safety and effectiveness of alglucosidase alfa (Lumizyme®) have not been established in individuals younger than one month of age.

Description

Pompe disease is a rare genetic disorder of glycogen metabolism that is caused by the absence or marked deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA). This disease is also known as glycogen storage disease type II, GSD II, glycogenosis type II, or acid maltase deficiency. GAA is necessary for proper muscle functioning and is used by the heart and muscle cells to convert a form of sugar called glycogen into energy. Without the GAA enzyme action, glycogen builds up in the cells of the heart, skeletal muscles, and hepatic tissues. Ultimately, these body organs are weakened by the intralysosomal accumulation of glycogen. Pompe disease encompasses a range of phenotypes, each including myopathy, but with significant variability in the age of onset, organ involvement, and clinical severity.

Infantile-onset Pompe disease occurs in an estimated 1 in every 40,000 to 300,000 births. Symptoms begin in the first months of life, with feeding problems, poor weight gain, muscle weakness, floppiness, and head lag. The primary symptom is heart and skeletal muscle weakness, which leads to the development of cardiomyopathy, progressing respiratory weakness, and death, usually from respiratory failure. Younger individuals generally have a much more aggressive form of the disease.

Juvenile/adult-onset Pompe disease results in intralysosomal accumulation of glycogen that is limited primarily to skeletal muscle, resulting in progressive muscle weakness. The onset can be as early as the first decade of childhood or as late as the sixth decade of adulthood. The primary symptom is muscle weakness progressing to respiratory weakness and death from respiratory failure over several years. The heart is usually spared. 

Enzyme replacement therapy has been shown to decrease heart size, maintain normal heart function, improve muscle function, tone, and strength, and reduce glycogen accumulation. Enzyme replacement therapy is currently not recommended for individuals with no symptoms or objective signs (proximal muscle weakness or reduced FVC in either upright or supine position) of Pompe disease.

Alglucosidase alfa (Lumizyme®) is FDA-approved for individuals with Pompe disease (GAA deficiency). The safety and efficacy was assessed in 57 treatment-naïve individuals with infantile-onset Pompe disease, aged 0.2 months to 3.5 years at first infusion, in three separate clinical trials. In all three trials ventilator-free survival improved significantly compared with an untreated historical control. The safety and efficacy of alglucosidase alfa (Lumizyme®) was also assessed in 90 individuals with juvenile/adult-onset Pompe disease in a randomized, double-blinded, placebo-controlled trial. Alglucosidase alfa (Lumizyme®) was shown to have a significant increase in forced vital capacity (FVC) and the distance an individual with juvenile/adult-onset Pompe disease can walk within 6 minutes (6 minute walk test).

There may be additional indications contained in the Policy section of this document due to evaluation of criteria highlighted in the Company’s off-label policy, and/or review of clinical guidelines issued by leading professional organizations and government entities.
References


American Hospital Formulary Service (AHFS). Drug Information 2014. Alglucosidase alfa. [Lexicomp Web site]. Updated 03/22/2018. Available at:http://online.lexi.com/lco/action/home[via subscription only]. Accessed January 11, 2019.

Cupler E, Berger K, et al. Consensus treatment recommendations for late-onset Pompe disease. Muscle Nerve. 2012;45(3):319-333.

Elsevier’s Clinical Pharmacology Compendium. Agalsidase alfa. [Clinical Pharmacology Web site]. 08/11/2014. Available at:http://online.lexi.com/lco/action/home#.. [via subscription only]. Accessed January 7, 2019.

Genzyme Corp. Alglucosidase alfa (Lumizyme®) [prescribing Information]. Cambridge, MA: Genzyme: 2014. Updated 08/2014. Available at: http://www.lumizyme.com/~/media/LumizymeUS/Files/lumizyme_prescribing_information.pdf Accessed January 11, 2019.

Kishnani PS, Corzo D, Nicolino M, et al. Recombinant human acid [alpha]-glucosidase: Major clinical benefits in infantile-onset Pompe disease. Neurology. 2007;68(2):99-109.

Kruer MC, Lysosomal Storage Disease.eMedicine[eMedicine Web site] updated 12/09/2015. Available at: http://emedicine.medscape.com/article/1182830-overview. Accessed January 11, 2019.

LaCana E, Yao LP, Pariser AR. The role of immune tolerance inductionin restoration of the efficacy of ERT in Pompe disease. Am J Med Genet C Semin Med Genet. 2012;160(1):30-9.

Leslie N, Bailey L GeneReviews® [Internet]. 08/31/2007; updated 05/11/2017. Available at: https://www.ncbi.nlm.nih.gov/books/NBK1261/#gsd2.Diagnosis. Accessed January 7, 2019.

Lexi-Drugs Compendium. Alglucosidase alfa. [Lexicomp Online Web site]. 01/01/19. Available at: http://online.lexi.com/lco/action/home [via subscription only]. Accessed January 11, 2019.

Micromedex®. Alglucosidase alfa. [Micromedex Web site]. 10/01/2018. Available at: http://www.micromedexsolutions.com/micromedex2/librarian/ [via subscription only]. Accessed January 11, 2019.

National Institute of Neurological Disorders and Stroke (NINDS). NINDS Pompe disease information page. [NINDS Web site]. Available at: http://www.ninds.nih.gov/disorders/pompe/pompe.htm. Accessed January 11, 2019.

Prater SN, Banugaria SG, Dearmey SM, et al. The emerging phenotype of long-term survivors with infantile Pompe disease. Genet Med. 2012;14(9):800-10.

US Food and Drug Administration (FDA). Alglucosidase alfa (Lumizyme®) SUPPLEMENT APPROVAL. Release REMS Requirement. [FDA Web site]. 08/01/2014. Available at: http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2014/125291Orig1s136ltr.pdf. Accessed January 11, 2019.

US Food and Drug Administration (FDA). Center for Drug Evaluation and Research. Alglucosidase alfa (Lumizyme®). Product approval information. [FDA Web site]. May 24, 2010. Available at: http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2010/125291s000ltr.pdf. Accessed January 11, 2019.

van der Ploeg A, Clemens P, et al. A randomized study of alglucosidase in late-onset Pompe's disease. New Engl J Med. 2010;362(15):1396-1406.






Coding

Inclusion of a code in this table does not imply reimbursement. Eligibility, benefits, limitations, exclusions, precertification/referral requirements, provider contracts, and Company policies apply.

The codes listed below are updated on a regular basis, in accordance with nationally accepted coding guidelines. Therefore, this policy applies to any and all future applicable coding changes, revisions, or updates.

In order to ensure optimal reimbursement, all health care services, devices, and pharmaceuticals should be reported using the billing codes and modifiers that most accurately represent the services rendered, unless otherwise directed by the Company.

The Coding Table lists any CPT, ICD-9, ICD-10, and HCPCS billing codes related only to the specific policy in which they appear.

CPT Procedure Code Number(s)

N/A


Professional and outpatient claims with a date of service on or before September 30, 2015, must be billed using ICD-9 codes. Professional and outpatient claims with a date of service on or after October 1, 2015, must be billed using ICD-10 codes.

Facility/Institutional inpatient claims with a date of discharge on or before September 30, 2015, must be billed with ICD-9 codes. Facility/Institutional inpatient claims with a date of discharge on or after October 1, 2015, must be billed with ICD-10 codes.


ICD - 10 Procedure Code Number(s)

N/A


Professional and outpatient claims with a date of service on or before September 30, 2015, must be billed using ICD-9 codes. Professional and outpatient claims with a date of service on or after October 1, 2015, must be billed using ICD-10 codes.

Facility/Institutional inpatient claims with a date of discharge on or before September 30, 2015, must be billed with ICD-9 codes. Facility/Institutional inpatient claims with a date of discharge on or after October 1, 2015, must be billed with ICD-10 codes.


ICD -10 Diagnosis Code Number(s)

E74.02 Pompe disease


HCPCS Level II Code Number(s)



MEDICALLY NECESSARY

J0221 Injection, alglucosidase alfa, (Lumizyme), 10 mg

NOT ELIGIBLE FOR REIMBURSEMENT

THE FOLLOWING CODE REPRESENTS ALGLUCLUCOSIDASE ALFA (MYOZYME®), WHICH IS NO LONGER MANUFACTURED AND HAS BEEN WITHDRAWN FROM THE MARKET:

J0220 Injection, alglucosidase alfa, 10 mg, not otherwise specified


Revenue Code Number(s)

N/A

Coding and Billing Requirements


Cross References


Policy History

Revisions from 08.00.72h
06/03/2019.This version of the policy will become effective 06/03/2019.

This policy has been updated to communicate dosing and frequency requirements for alglucosidase alfa (e.g., Lumizyme®) and requirements for laboratory and/or genetic testing consistent with the US Food and Drug Administration (FDA) labeling.

Revisions from 08.00.72g
06/06/2018This policy has been reissued in accordance with the Company's annual review process.
01/22/2018This version of the policy will become effective 01/22/2018.

This policy has been updated to be consistent with the US Food and Drug Administration (FDA) labeling and NCCN compendia.

This policy was updated to remove alglucosidase alfa (Myozyme®), which was discontinued in 2013. The title was also changed to remove "Myozyme".


Effective 10/05/2017 this policy has been updated to the new policy template format.


Version Effective Date: 06/03/2019
Version Issued Date: 06/03/2019
Version Reissued Date: N/A

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