Lysosomal Storage Disorders

Home , Alglucosidase alfa, Asfotase alfa, Idursulfase, Sebelipase alfa

Number: RX501.067

Effective Date: 01-15-2019

Coverage:

*CAREFULLY CHECK STATE REGULATIONS AND/OR THE MEMBER CONTRACT*

Medical policies are a set of written guidelines that support current standards of practice. They are based on current peer-reviewed scientific literature. A requested therapy must be proven effective for the relevant diagnosis or procedure. For drug therapy, the proposed dose, frequency and duration of therapy must be consistent with recommendations in at least one authoritative source. This medical policy is supported by FDA- approved labeling and nationally recognized authoritative references. These references include, but are not limited to: MCG care guidelines, DrugDex (IIb strength of recommendation or higher), NCCN Guidelines (IIb level of evidence or higher), NCCN Compendia (IIb level of evidence or higher), professional society guidelines, and CMS coverage policy.

Enzyme-replacement therapy when utilized for the treatment of lysosomal storage disorders (LSD) may be considered medically necessary for the specific indications noted under each of the following enzyme- replacements therapies:

I. Agalsidase beta (Fabrazyme®)

Agalsidase beta (Fabrazyme) may be considered medically necessary for the use in members when the patient has a diagnosis of Fabry disease.

Agalsidase beta (Fabrazyme) is considered experimental, investigational and/or unproven for the treatment of all other indications.

II. Idursulfase (Elaprase™)

Idursulfase (Elaprase™) may be considered medically necessary when:

 The patient is 5 years of age or greater; AND

• The patient has a diagnosis of Hunter’s syndrome (mucopolysaccharidosis II [MPS II]).

Idursulfase (Elaprase™) is considered experimental, investigational and/or unproven for the treatment of all other indications.

III. Alglucosidase alfa (Myozyme®)

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© Copyright Prime Therapeutics LLC. 01/2021 All Rights Reserved Effective 1/1/2021 Alglucosidase alfa (Myozyme®) may be considered medically necessary for the treatment of infantile-onset Pompe disease (acid α-glucosidase [GAA] deficiency).

Alglucosidase alfa (Myozyme®) is considered experimental, investigational and/or unproven for the treatment of all other indications, including but not limited to treatment of persons with other forms of Pompe disease.

IV. Alglucosidase alfa (Lumizyme™)

Alglucosidase alfa (Lumizyme™) may be considered medically necessary for patients with Pompe disease (GAA deficiency).

Alglucosidase alfa (Lumizyme™) is considered experimental, investigational and/or unproven for the treatment of all other indications.

V. Asfotase alfa (Strensiq™)

Asfotase alfa (Strensiq™) may be considered medically necessary for the treatment of patients with perinatal/infantile-and juvenile-onset hypophosphatasia (HPP).

Asfotase alfa (Strensiq™) is considered experimental, investigational and/or unproven for all other indications.

VI. Elosulfase alfa (Vimizim™)

Elosulfase alfa (Vimizim™) may be considered medically necessaryforpatients 5 years of age or older with a documented clinical diagnosis of mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).

Elosulfase alfa (Vimizim™) is considered experimental, investigational and/or unproven for all other indications.

VII. Naglazyme® (Galsulfase)

Naglazyme® (Galsulfase) may be considered medically necessary for patients with Mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome).

Naglazyme® (Galsulfase) is considered experimental, investigational and/or unproven for all other indications.

VIII. Imiglucerase (Cerezyme®)

Imiglucerase (Cerezyme®) may be considered medically necessary for long-term enzyme replacement therapy (ERT) for patients 2 years of age or greater with a confirmed diagnosis of Type 1 Gaucher disease that results in one or more of the following conditions:

 Anemia; or

 Bone disease; or

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• Thrombocytopenia.

Imiglucerase (Cerezyme®) is considered experimental, investigational and/or unproven for all other indications.

IX. Laronidase (Aldurazyme®)

Laronidase (Aldurazyme®) may be considered medically necessary for patients 6 months of age or greater with one of the following:

 Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I); or

• Scheie form who have moderate to severe symptoms.

Laronidase (Aldurazyme®) is considered experimental, investigational and/or unproven for all other indications, including but not limited to treatment of members with the Scheie form of MPS I who have mild symptoms.

X. Sebelipase alfa (Kanuma™)

Sebelipase alfa (Kanuma™) may be considered medically necessary when:

• The patient is one month of age or greater; AND

• The patient has a diagnosis of lysosomal acid lipase (LAL) deficiency (i.e. Wolman’s Disease).

Sebelipase alfa (Kanuma™) is considered experimental, investigational and/or unproven for all other indications.

XI. Taliglucerase alfa (Elelyso™)

Taliglucerase alfa (Elelyso™) may be considered medically necessary for long-term ERT for patients 4 years of age or greater with a confirmed diagnosis of Type 1 Gaucher disease.

Taliglucerase alfa (Elelyso™) is considered experimental, investigational and/or unproven for all other indications.

XII. Velaglucerase alpha (VPRIV)

Velaglucerase alpha (VPRIV) may be considered medically necessary for long-term ERT for patients 4 years of age or greater with type 1 Gaucher disease.

Velaglucerase alpha (VPRIV) is considered experimental, investigational and/or unproven for all other indications.

XIII. Vestronidase alfa-vjbk (MepseviiTM)

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© Copyright Prime Therapeutics LLC. 01/2021 All Rights Reserved Effective 1/1/2021 Vestronidase alfa-vjbk (MepseviiTM) may be considered medically necessary in patients 5 months of age or greater with a diagnosis of Mucopolysaccharidosis VII (MPS VII, Sly syndrome).

Vestronidase alfa-vjbk (MepseviiTM) is considered experimental, investigational and/or unproven for all other indications.

Length of Approval: 12 months

TARGET AGENTS Agent GPI HCPCS Code Multisource Code Aldurazyme (laronidase) 2.9 mg / 5 mL injection 30906550002020 J1931 M, N, O, or Y Cerezyme (imiglucerase) 400 Unit injection 82700050002120 J1786 M, N, O, or Y Elaprase (idursulfase) 6 mg / 3 mL injection 30906850002020 J1743 M, N, O, or Y Elelyso (taliglucerase alfa) 200 unit injection 82700080102120 J3060 M, N, O, or Y Fabrazyme (agalsidase beta) 5 mg injection 30903610102110 J0180 M, N, O, or Y 35 mg injection 30903610102120 J0180 M, N, O, or Y Kanuma (sebelipase alfa) 20 mg / 10 mL injection 30906360002020 J2840 M, N, O, or Y Lumizyme (alglucosidase alfa) 50 mg injection 30907715002120 J0221 M, N, O, or Y Mepsevii (vestronidase alfa-vjbk) 10 mg / 5 mL injection 30907680202020 J3397 M, N, O, or Y Naglazyme (galsulfase) 1 mg / 1 mL injection 30907535002020 J1458 M, N, O, or Y Vimizim (elosulfase alfa) 5 mg / 5 mL injection 30907030052020 J1322 M, N, O, or Y Vpriv (velaglucerase alfa) 400 unit injection 82700085102120 J3385 M, N, O, or Y

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