POLICY NUMBER

S.5.01.526

Aldurazyme (laronidase)

Cerezyme (imiglucerase)

Elaprase (idursulfase)

Elelyso (taliglucerase)

Elfabrio (pegunigalsidase alfa)

Fabrazyme (agalsidase)

Kanuma (sebelipase alfa)

Lamzede (velmanase alfa-tycv)

Lumizyme (alglucosidase)

Mepsevii (vestronidase alfa-vjbk)

Naglazyme (galsulfase)

Nexviazyme (avalglucosidase alfa-ngpt)

Pombiliti (cipaglucosidase alfa-atga)

Vimizim (elosulfase alfa)

Vpriv (velaglucerase)

Xenpozyme (olipudase alfa-rpcp)

Please perform a search of the State Health Plan Medical Drug Formulary for drugs administered and billed through the medical setting.

DESCRIPTION

Lysosomal storage disorders (LSDs) are a group of inherited metabolic disorders in which harmful amounts of fatty materials accumulate in various cells and tissues in the body. People with these disorders either do not produce enough of one of the enzymes needed to metabolize or they produce enzymes that do not work properly. Although there is no cure, enzyme replacement therapy is available for some of the LSDs.

POLICY

The use of samples by an individual will not be considered current or stable therapy to satisfy Medical Policy requirements.

The following medications are not covered on the State Health Plan Medical Drug Formulary:

• Elfabrio (pegunigalsidase alfa)

• Pombiliti (cipaglucosidase alfa-atga)

• Xenpozyme (olipudase alfa-rpcp)

• Lamzede (velmanase alfa-tycv)

Initial Criteria

The requested medication may be considered medically necessary when ALL of the following criteria are met:

1. ONE of the following:

   1. The individual has a diagnosis of Gaucher disease type 1 and ALL of the following:

      1. The individual’s diagnosis is established by ONE of the following:

         1. Demonstration of deficient beta-glucocerebrosidase activity in leukocytes or fibroblasts; OR

         2. Genotype testing indicates a mutation of 2 alleles of the glucocerebrosidase genome;

      2. The individual has at least ONE of the following clinical presentations at baseline (prior to therapy for the requested indication):

         1. Anemia defined as mean hemoglobin (Hb) level below the testing laboratory’s lower limit of normal range based on age and gender;

         2. Thrombocytopenia (platelet count less than 100,000 microliter on at least 2 measurements);

         3. Hepatomegaly;

         4. Splenomegaly;

         5. Growth failure (i.e., growth velocity is below the standard mean for age); OR

         6. Evidence of bone disease with other causes ruled out;

      3. The individual does NOT have any neuropathic symptoms indicative of Gaucher disease type 2 or type 3 [e.g., bulbar signs (e.g., stridor, strabismus, swallowing difficulty), pyramidal signs (e.g., opisthotonos, head retroflexion, spasticity, trismus), oculomotor apraxia, tonic-clonic seizures, myoclonic epilepsy, dementia, ataxia]; AND

      4. ONE of the following:

         1. The request is for Cerezyme (imiglucerase); OR

         2. The request is for Elelyso (taliglucerase) or Vpriv (velaglucerase), and the individual is 4 years of age or older;

   2. The individual has a diagnosis of mucopolysaccharidoses and ONE of the following:

      1. The request is for Aldurazyme (laronidase) and ALL of the following:

         1. The individual has a documented diagnosis of mucopolysaccharidosis I (MPS I) confirmed by ONE of the following:

            1. Deficiency of alpha-L-iduronidase enzyme (IDUA) activity; OR

            2. Detection of biallelic pathogenic mutations in the IDUA gene by molecular genetic testing;

         2. The individual has ONE of the following:

            1. Hurler form (i.e., severe MPS I);

            2. Hurler-Scheie form (i.e., attenuated MPS I); OR

            3. Scheie form (i.e., attenuated MPS I) with moderate to severe symptoms (e.g., normal intelligence, less progressive physical problems, corneal clouding, joint stiffness, valvular heart disease);

         3. The individual demonstrates clinical signs and symptoms of the disease (e.g., asymptomatic with affected older sibling, cardiac abnormalities, corneal clouding, dysostosis multiplex, hepatomegaly, restrictive lung disease, etc.); AND

         4. Documented baseline value for urinary glycosaminoglycan (uGAG) has been obtained;

      2. The request is for Elaprase (idursulfase) and ALL of the following:

         1. The individual has a documented diagnosis of Hunter syndrome (mucopolysaccharidosis II, MPS II) confirmed by ONE of the following:

            1. Deficiency of iduronate-2-sulfatase (IDS) enzyme activity in white cells, fibroblasts, or plasma in the presence of normal activity of at least one other sulfatase; OR

            2. Detection of pathogenic mutations in the IDS gene by molecular genetic testing;

         2. The individual is 16 months of age or older;

         3. The individual demonstrates clinical signs and symptoms of the disease (e.g., hepatosplenomegaly, skeletal deformities, dysostosis, neurocognitive decline, cardiovascular disorders, etc.); AND

         4. Documented baseline value for urinary glycosaminoglycan (uGAG) has been obtained;

      3. The request is for Mepsevii (vestronidase alfa) and ALL of the following:

         1. The individual has a diagnosis of mucopolysaccharidosis VII (MPS VII, Sly Syndrome) confirmed by BOTH of the following:

            1. Beta-glucuronidase enzyme deficiency in peripheral blood leukocytes, fibroblasts, or dried blood spots; AND

            2. Detection of pathogenic mutations in the GUSB gene by molecular genetic testing; AND

         2. Presence of clinical signs and symptoms of the disease (e.g., enlarged liver and spleen, joint limitations, airway obstruction or pulmonary problems, limitation of mobility while still ambulatory, etc.);

      4. The request is for Naglazyme (galsulfase) and ALL of the following:

         1. The individual has a documented diagnosis of mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome) confirmed by ONE of the following:

            1. Deficiency of N-acetylgalactosamine 4-sulfatase (arylsulfatase B) enzyme activity AND elevated urinary glycosaminoglycan (uGAG) level (i.e. dermatan sulfate or chondroitin sulfate) defined as being above the upper limit of normal by the reference laboratory; OR

            2. Detection of pathogenic mutations in the ARSB gene by molecular genetic testing; AND

         2. The individual demonstrates clinical signs and symptoms of the disease (e.g., hepatosplenomegaly, skeletal deformities, dysostosis, neurocognitive decline, cardiovascular disorders, etc.); OR

      5. The request is for Vimizim (elosulfase alfa) and ALL of the following:

         1. The individual has a diagnosis of mucopolysaccharidosis type IVA (Morquio A syndrome) confirmed by ONE of the following:

            1. Absence or marked reduction in N-acetylgalactosamine 6-sulfatase (GALNS) enzyme activity in cultured fibroblasts or leukocytes; OR

            2. Detection of biallelic pathogenic mutations in the GALNS gene by genetic molecular testing (i.e., sequence analysis and/or deletion/duplication analysis);

         2. The individual is 5 years of age or older; AND

         3. Presence of clinical signs and symptoms of the disease (e.g., kyphoscoliosis, c, pectus carinatum, gait disturbance, growth deficiency, etc.);

   3. The individual has a diagnosis of Fabry disease and ALL of the following:

      1. The request is for Fabrazyme (agalsidase beta);

      2. The individual is 2 years of age or older;

      3. The diagnosis is confirmed by at least ONE of the following:

         1. Absence or deficiency (< 5% of mean) of normal alpha-galactosidase A (α-Gal A) enzyme activity in leukocytes, dried blood spots, or serum analysis (males only); OR

         2. Detection of pathogenic mutations in the GLA gene by molecular genetic testing; AND

      4. The individual has a baseline of at least ONE of the following:

         1. Tissue globotriaosylceramide (Gb3/GL-3) inclusions;

         2. Plasma or urinary globotriaosylceramide (Gb3/GL-3) or globotriaosylsphingosine (lyso-Gb3); OR

         3. Clinical signs and/or symptoms of disease (e.g., dermatologic, gastrointestinal, pulmonary, vascular, renal, cardiac, neurologic manifestations);

   4. The individual has a diagnosis of lysosomal acid lipase deficiency (LAL-D) and ALL of the following:

      1. The request is for Kanuma (sebelipase alfa);

      2. The diagnosis is confirmed by at least ONE of the following:

         1. Absence or deficiency lysosomal acid lipase (LAL) enzyme activity in peripheral blood leukocytes, fibroblasts, or dried blood spots; OR

         2. Detection of biallelic pathogenic variants in the lipase A, lysosomal acid type (LIPA) gene; AND

      3. Documented baseline values for one or more of the following have been obtained:

         1. Weight-for-age z-scores for patients exhibiting growth failure;

         2. Low-density lipoprotein cholesterol (LDL-c);

         3. High-density lipoprotein cholesterol (HDL-c);

         4. Non-HDL-c;

         5. Triglycerides; OR

         6. Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT); OR

   5. The individual has a diagnosis of Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) and ALL of the following:

      1. The diagnosis is confirmed by at least ONE of the following:

         1. Deficiency of acid alpha-glucosidase (GAA) enzyme activity; OR

         2. Detection of biallelic pathogenic variants in the GAA gene by molecular genetic testing;

      2. Documented baseline values/status for one or more of the following have been obtained:

         1. Motor function (e.g. PEDI-Pompe scale);

         2. Neurologic function;

         3. Percent-predicted forced vital capacity (FVC);

         4. Walking distance or 6-minute walk test (6MWT); OR

         5. Cardiac involvement (e.g., echocardiogram); AND

      3. ONE of the following:

         1. The request is for Lumizyme (alflucosidase); OR

         2. The request is for Nexviazyme (avalglucosidase alfa-ngpt) and BOTH of the following;

            1. The individual is 1 year of age or older; AND

            2. The individual has late-onset Pompe disease;

2. If the request is for enzyme replacement therapy, the requested agent will not be used in combination with other enzyme replacement therapy;

3. The prescriber is a specialist or has consulted with a specialist in the area of the individual's diagnosis (e.g. endocrinologist, geneticist, hematologist);

4. The prescribed dosage is within the program quantity limits based on FDA approved labeled dosage; AND

5. The individual does not have any FDA-labeled contraindication(s) to the requested agent.

Length of Approval: 12 months 

Renewal Criteria

The requested agent may be approved for RENEWAL when ALL of the following criteria are met:

1. The individual has previously been approved for the requested agent through the BCBSMS review process;

2. The individual has demonstrated a beneficial response to therapy compared to pretreatment age-appropriate baseline values in one or more of the following:

   1. Gaucher disease Type 1: reduced severity or resolution of anemia, thrombocytopenia, bone disease, hepatomegaly, and/or splenomegaly;

   2. Mucopolysaccharidosis (MPS I, MPS II [Hunter Syndrome], MPS VI [Maroteaux-Lamy syndrome]): stability or improvement in cardiac status, upper airway obstruction during sleep, growth velocity, mental development, FVC and/or 6-MWT, increased joint range of motion, decreased left ventricular hypertrophy, and/or documented reduction in uGAG levels;

   3. Fabry disease: reduction or stabilization in plasma or urinary globotriaosylceramide (Gb3/GL-3) or globotriaosylsphingosine (lyso-Gb3) and/or improvement or stabilization of dermatologic, gastrointestinal, pulmonary, vascular, renal, cardiac, or neurologic manifestations;

   4. Lysosomal acid lipase (LAL) deficiency: improvement in weight-for-age z-scores for patients exhibiting growth failure, LDL-c, HDL-c, non-HDL-c, triglycerides, AST and/or ALT;

   5. Infantile-onset Pompe disease: stabilization or improvement in muscle weakness, motor function, respiratory function, cardiac involvement, FVC and/or 6-MWT; OR

   6. Late-onset (non-infantile) Pompe disease: stabilization or improvement in FVC and/or 6-MWT;

3. The prescriber is a specialist or has consulted with a specialist in the area of the individual's diagnosis (e.g., endocrinologist, geneticist, hematologist);

4. The prescribed dosage is within the program quantity limits based on FDA approved labeled dosage; AND

5. The individual does not have any FDA-labeled contraindication(s) to the requested agent.

Length of Approval: 12 months

POLICY EXCEPTIONS

State Health Plan (State and School Employees): Self-administered drugs may be covered under a prescription drug benefit plan administered by the State Health Plan’s Pharmacy Benefit Manager. Please perform a formulary drug search at https://www.dfa.ms.gov/cvs-caremark and submit any required Prior Authorization Requests for coverage determination to the Plan’s Pharmacy Benefit Manager. Services related to delivery and/or administration of a self-administered drug are not covered under the medical benefit.

POLICY GUIDELINES

The coverage guidelines outlined in the Medical Policy Manual should not be used in lieu of the Participant's specific benefit plan language.

Medically Necessary is defined as those services, treatments, procedures, equipment, drugs, devices, items or supplies furnished by a covered Provider that are required to identify or treat a Participant's illness, injury or Mental Health Disorders, and which Company determines are covered under this Benefit Plan based on the criteria as follows in A through D:

A. consistent with the symptoms or diagnosis and treatment of the Participant's condition, illness, or injury; and

B. appropriate with regard to standards of good medical practice; and

C. not solely for the convenience of the Participant, his or her Provider; and

D. the most appropriate supply or level of care which can safely be provided to Participant. When applied to the care of an Inpatient, it further means that services for the Participant's medical symptoms or conditions require that the services cannot be safely provided to the Participant as an Outpatient.

For the definition of medical necessity, “standards of good medical practice” means standards that are based on credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community, and physician specialty society recommendations, and the views of medical practitioners practicing in relevant clinical areas and any other relevant factors. BCBSMS makes no payment for services, treatments, procedures, equipment, drugs, devices, items or supplies which are not documented to be Medically Necessary. The fact that a Physician or other Provider has prescribed, ordered, recommended, or approved a service or supply does not in itself, make it Medically Necessary.

BCBSMS determines patient medication trial and adherence by a review of pharmacy claims data over the preceding twelve months. Additional information may be requested on a case-by-case basis to allow for proper review. If individual is new to BCBSMS and pharmacy records are needed to confirm medication trials and adherence, it is the responsibility of the individual and/or requesting provider to obtain said records and to submit them to BCBSMS upon request. Medical records from the provider that list previously prescribed medications will not be sufficient to show medication trials or adherence.

POLICY HISTORY

10/01/2024: Created separate State and School Employees' Health Insurance Plan - Lysosomal Storage Disorders policy. Coverage criteria unchanged.

01/15/2025: Policy updated to state that Pombiliti (cipaglucosidase alfa-atga) is considered not medically necessary as there are other formulary alternatives covered by the Plan for the treatment of Pompe disease. Sources updated. Code Reference section updated to add HCPCS code J1203 to the Not Medically Necessary Codes table.

06/03/2025: Policy reviewed by Pharmacy & Therapeutics (P&T) Committee; no changes.

09/15/2026: Policy reviewed. Policy statement revised to state that the use of samples by an individual will not be considered current or stable therapy to satisfy Medical Policy requirements. Added policy statement to list Elfabrio (pegunigalsidase alfa), Pombiliti (cipaglucosidase alfa-atga), Xenpozyme (olipudase alfa-rpcp), and Lamzede (velmanase alfa-tycv) as medications not covered on the State Health Plan Medical Drug Formulary. Medically necessary criteria extensively revised and updated to add coverage criteria for Mepsevii (vestronidase alfa) and Vimizim (elosulfase alfa). Revised renewal criteria. Sources updated. Code Reference section updated to add HCPCS codes J1322 and J3397 to the Medically Necessary Codes table. Added HCPCS codes J0217 and J0218 to the Not Medically Necessary Codes table.

SOURCE(S)

1. Aldurazyme prescribing information. Genzyme Corporation. July 2026. Last accessed September 2026.

2. Cerezyme prescribing information. Genzyme Corporation. August 2026. Last accessed September 2026.

3. Elaprase prescribing information. Takeda Pharmaceuticals America, Inc. April 2025. Last accessed September 2026.

4. Elelyso prescribing information. Pfizer Laboratories Div Pfizer Inc. December 2025. Last accessed September 2026.

5. Elfabrio prescribing information. Chiesi USA, Inc. July 2024. Last accessed September 2026.

6. Fabrazyme prescribing information. Genzyme Corporation. August 2025. Last accessed September 2026.

7. Kanuma prescribing information. Alexion Pharmaceuticals, Inc. July 2025. Last accessed September 2026.

8. Lamzede prescribing information. Chiesi USA, Inc. February 2023. Last accessed April 2026.

9. Lumizyme prescribing information. Genzyme Corporation. January 2025. Last accessed September 2026.

10. Mepsevii prescribing information. Ultragenyx Pharmaceutical Inc. November 2025. Last accessed April 2026.

11. Naglazyme prescribing information. BioMarin Pharmaceutical Inc. September 2024. Last accessed September 2026.

12. Nexviazyme prescribing information. Genzyme Corporation. June 2025. Last accessed September 2026.

13. Pombilti prescribing information. AMICUS THERAPEUTICS US, LLC. August 2026. Last accessed September 2026.

14. Vimizim prescribing information. BioMarin Pharmaceutical Inc. October 2025. Last accessed April 2026.

15. Vpriv prescribing information. Takeda Pharmaceuticals America, Inc. November 2024. Last accessed September 2026.

16. Xenpozyme prescribing information. Genzyme Corporation. December 2025. Last accessed April 2026.

CODE REFERENCE

This may not be a comprehensive list of procedure codes applicable to this policy.

The code(s) listed below are ONLY medically necessary if the procedure is performed according to the "Policy" section of this document.

Medically Necessary Codes

Not Medically Necessary Codes

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