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Coverage criteria policies

Mucopolysaccharidoses (MPS) Drug Therapy

These services may or may not be covered by all HealthPartners plans. Please see your plan documents for your own coverage information. If there is a difference between this general information and your plan documents, your plan documents will be used to determine your coverage.

Administrative Process

Enzyme replacement therapy for mucopolysaccharidoses (MPS) requires prior authorization from Pharmacy Administration. The setting of drug administration will be reviewed as part of the prior authorization.

Note:

For all inquiries, transfer to the Medical Injectable Line (ext 26135).

Coverage

Enzyme replacement therapy for MPS is generally covered when:

    1. Prescribed by a specialist; and,

    2. Prescribed for an FDA-approved indication using an approved regimen as below. A current patient weight is required for all requests; and,

    3. The patient and/or guardian has attested that they will adhere to the treatment plan; and,

    4. For commercial products only (does not apply to Medicare or Minnesota Health Care Programs products), medication administration must occur at a clinic office or home-infusion setting unless medical necessity is met based on the criteria below, supported by medical documentation:

    1. The patient has experienced a severe or life-threatening reaction with previous infusions of the same or similar products; or,
    2. The patient has a medical condition that renders him or her unstable, exceptionally complex, immunocompromised or otherwise high-risk such that continued oversight in the current facility is required; or,
    3. There are no alternative settings available to the patient as a result of both of the following:
      1. The patient is unable to use home-infusion services as documented by the physician, social worker, or infusion provider; AND,
      2. The patient is unable to access alternative settings due to unreasonable distance [>30 miles] or other extenuating circumstances.

Initial approvals will be for twelve months.

Annual reauthorizations will require medical chart documentation that the patient has been seen within the past 12 months and that markers of disease such as pulmonary function, spleen volume, walking or stair-climbing capacity are improved by therapy.

Indications and regimens that are covered

Aldurazyme (laronidase) is generally covered when prescribed for patients with Hurler and Hurler-Scheie forms of MPS I and for patients with the Scheie form who have moderate to severe symptoms. The approved regimen is 0.58 mg/kg of body weight administered once weekly as an intravenous (IV) infusion.

Elaprase (idursulfase) is indicated for patients with Hunter syndrome (MPS II). The approved regimen is 0.5 mg per kg of body weight administered once every week as an intravenous infusion.

Naglazyme (galsulfase) is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for patients with MPS VI (Maroteaux-Lamy syndrome). The approved dosage regimen is 1 mg per kg of body weight administered once weekly as an intravenous infusion.

Vimizim (elosulfase) is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA: Morquio A syndrome). The FDA approved regimen is 2 mg per kg body weight administered once every week as an intravenous infusion over a minimum of 3.5 to 4.5 hours, based on infusion volume.

Indications that are not covered

Requests for use in indications that are not FDA-approved will be reviewed on a case by case basis for medical necessity.

Definitions

Mucopolysaccharidoses (MPS) is a term for several lysosomal storage disorders caused by the deficiency of enzymes required for the breakdown of glycosaminoglycans, also known as mycopolysaccharides. Enzyme replacement therapy is approved for patients with MPS I, MPS II, and MPS VI. MPS I or Hurler, Hurler-Scheie and Scheie syndromes result from a deficiency of alpha-L-iduronidase or laronidase.

Aldurazyme (laronidase) is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for patients with Hurler and Hurler-Scheie forms of MPS I and for patients with the Scheie form who have moderate to severe symptoms. The risks and benefits of treating mildly affected patients with the Scheie form have not been established. Aldurazyme has been shown to improve pulmonary function and walking capacity. It has not been evaluated for effects on the central nervous system manifestations of the disorder.

MPS II or Hunter syndrome results from insufficient levels of iduronate sulfatase or idursulfase.

Elaprase (idursulfase) is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for patients with Hunter syndrome (Mucopolysaccharidosis II, MPS II). ELAPRASE has been shown to improve walking capacity in patients 5 years and older. In patients 16 months to 5 years of age, no data are available to demonstrate improvement in disease-related symptoms or long term clinical outcome; however, treatment with ELAPRASE has reduced spleen volume similarly to that of adults and children 5 years of age and older. The safety and efficacy of ELAPRASE have not been established in pediatric patients less than 16 months of age (1).

MPS VI or Maroteaux-Lamy syndrome results from a deficiency of N-acetylgalactosamine-4-sulfate or galsulfase.

Naglazyme (galsulfase) is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for patients with MPS VI (Maroteaux-Lamy syndrome). It has been shown to improve walking and stair-climbing capacity.

Elosulfase (Vimizim) is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA: Morquio A syndrome).

Setting: The type of physical site where the drug is provided. Settings include inpatient hospital, outpatient hospital, clinic office, or home-infusion.

  • Outpatient Hospital sites have physicians and practitioners on-site and are the appropriate site to manage unstable patients and patients experiencing certain moderate to severe adverse events. Hospital settings are typically the highest-cost, most-intensive, and are the highest level settings.
  • Clinic offices are lower level settings which are not outpatient hospital settings that can manage some unstable patients and patients experiencing adverse events. Physicians may or may not be readily available.
  • Home-infusion is a lower level setting, and is performed by a licensed nurse supported by a licensed pharmacy who have expertise in administering complex medications in a patient’s home. Home infusion providers regularly manage mild to moderate adverse events, and are prepared to manage severe adverse events if needed.

If available, codes are listed below for informational purposes only, and do not guarantee member coverage or provider reimbursement. The list may not be all-inclusive.

HCPCS Codes

Codes

Description

J1931

Injection, laronidase, 0.1 mg (Aldurazyme)

J1743

Injection, idursulfase, 1 mg (Elaprase)

J1458

Injection, galsulfase, 1 mg (Naglazyme)

J1322

Injection, elosulfase alfa, 1mg (Vimizim)

   
NDC Codes

Codes

Description

58468007001

Aldurazyme 2.9 mg/ 5mL single use vial

54092070001

Elaprase 6 mg/ 3 mL single use vial

68135002001

Naglazyme 1 mg/mL single use vial

68135010001

Vimizim 5 mg / 5 mL single use vial

CPT Copyright American Medical Association. All rights reserved. CPT is a registered trademark of the American Medical Association.

Products

This information is for most, but not all, HealthPartners plans. Please read your plan documents to see if your plan has limits or will not cover some items. If there is a difference between this general information and your plan documents, your plan documents will be used to determine your coverage. These coverage criteria may not apply to Medicare Products if Medicare requires different coverage. For more information regarding Medicare coverage criteria or for a copy of a Medicare coverage policy, contact Member Services at 952-883-7979 or 1-800-233-9645.

References

  1. Elaprase prescribing information. Shire Human Genetic Therapies, Inc. June 2013.
  2. Aldurazyme prescribing information. BioMarin/Genzyme LLC. April 2013.
  3. Naglazyme prescribing information. BioMarin Pharmaceutical Inc. March 2013.
  4. Vimizim prescribing information. BioMarin Pharmaceuticals Inc. February 2014.

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Policy activity

  • 11/14/2011 - Date of origin
  • 01/01/2017 - Effective date
Review date
  • 11/2016
Revision date
  • 10/01/2016

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