FDA-approved, for example: 

• Adenosine deaminase severe combined immunodeficiency (ADA-SCID) (Adagen, Revcovi)
• Fabry disease (Fabrazyme, Galafold)
• Gaucher Disease Type 1 (Cerdelga, Cerezyme, Elelyso, miglustat, Vpriv)
• Hunter Syndrome (Elaprase)
• Hyperammonemia due to the deficiency of the hepatic enzyme N-acetylglutamate synthetase (NAGS) (Carbaglu)
• Hypophosphatasia (Strensiq)
• Lysosomal acid lipase deficiency (Kanuma)
• Mucopolysaccharidosis I (Aldurazyme)
• Mucopolysaccharidosis IVA (Morquio A syndrome) (Vimizim)
• Mucopolysaccharidosis VI (Naglazyme)
• Mucopolysaccharidosis VII (Sly syndrome) (Mepsevii)
• Phenylketonuria (Kuvan, Palynziq)
• Pompe disease (Lumizyme)
• Urea cycle disorder (Ravicti)

Note: The above list may not include all FDA-approved indications.

Please note: In the case where the prior authorization (PA) status column indicates PA, both the brand and generic (if available) require PA. Typically, the generic is preferred when available unless the brand-name drug appears on the MassHealth Brand Name Preferred Over Generic Drug List. In general, when requesting the non-preferred version, whether the brand or generic, the prescriber must provide medical records documenting an inadequate response or adverse reaction to the preferred version, in addition to satisfying the criteria for the drug itself.

• All PA requests must include clinical diagnosis, drug name, dose, and frequency.
• A preferred drug may be designated for this therapeutic class. In general, MassHealth requires a trial of the preferred drug or clinical rationale for prescribing a non-preferred drug within a therapeutic class. Additional information about these agents, including PA requirements and preferred products, can be found within the MassHealth Drug List at www.mass.gov/druglist.
• Additional criteria may apply, depending upon the member’s condition and requested medication (see below).

Adagen

• Documentation of all of the following is required:
  • appropriate diagnosis; and
  • laboratory results documenting one of the following:
    • absent ADA enzymatic activity in lysed erythrocytes; or 
    • elevated levels of adenosine and deoxyadenosine in the urine and plasma; or 
    • a marked increase in deoxyadenosine triphosphate (dATP) levels in erythrocyte lysates; or 
    • a significant decrease in ATP concentration in red blood cells; or 
    • absent or extremely low levels of N adenosylhomocysteine hydrolase in red blood cells; or 
    • severe T cell deficiency manifested by lymphopenia and poor T cell responses to mitogens and antigens; or 
    • absent thymic shadow on chest radiograph; and
  • member's current weight.

Aldurazyme

• Documentation of all of the following is required:
  • appropriate diagnosis; and
  • results from an enzyme assay test showing reduced lysosomal alpha-L-iduronidase activity in peripheral blood leukocytes, plasma, or cultured fibroblasts; and
  • member's current weight.

Carbaglu

• Documentation of all of the following is required:
  • appropriate diagnosis; and
  • appropriate dosing; and
  • results from genetic test or an enzyme assay test supporting the diagnosis.

Cerdelga

• Documentation of all of the following is required:
  • appropriate diagnosis; and
  • member is ≥ 18 years of age; and
  • results from enzyme assay test showing reduced activity of glucocerebrosidase; and
  • member is not currently receiving enzyme replacement therapy.

Cerezyme and Vpriv

• Documentation of all of the following is required:
  • appropriate diagnosis; and
  • results from an enzyme assay test showing reduced activity of the enzyme glucocerebrosidase; and
  • member's current weight.

Elaprase

• Documentation of all of the following is required:
  • appropriate diagnosis; and
  • results from an iduronate-2-sulfatase assay test showing reduced or absent activity in the serum, white blood cells, or fibroblasts; and
  • member's current weight.

Elelyso

• Documentation of all of the following is required:
  • appropriate diagnosis; and
  • results from an enzyme assay test showing reduced activity of the enzyme glucocerebrosidase; and
  • member is ≥ 18 years of age; and
  • member's current weight.

Fabrazyme

• Documentation of all of the following is required:
  • appropriate diagnosis; and
  • results from an enzyme assay test showing reduced or absent α-galactosidase A (α-GAL) enzyme activity in plasma, leukocytes, tears, or biopsied tissue; and
  • member's current weight.

Galafold

• Documentation of all of the following is required:
  • appropriate diagnosis; and
  • member is ≥ 18 years of age; and
  • prescriber is a specialist (e.g., clinical genetics specialist or nephrologist) or consult is provided; and
  • results from an enzyme assay test showing reduced or absent α-galactosidase A (α-GAL) enzyme activity in plasma, leukocytes, tears, or biopsied tissue; and
  • member has GLA variants which are amenable to treatment with the requested agent; and
  • request is within quantity limit of 15 units/30 days.

Kanuma

• Documentation of all of the following is required:
  • diagnosis of lysosomal acid lipase deficiency; and
  • member's current weight.

Kuvan

• Documentation of all of the following is required:
  • appropriate diagnosis; and
  • results from genetic testing or molecular analysis to confirm diagnosis; and
  • documentation that medication will be used in conjunction with a phenylalanine-restricted diet; and
  • member's current weight.

Lumizyme

• Documentation of all of the following is required:
  • appropriate diagnosis; and
  • results from acid maltase enzyme alpha-glucosidase (GAA) assay test showing reduced or absent activity from cultured skin fibroblasts, muscle biopsy, or lymphocyte testing; and
  • member's current weight.

Mepsevii

• Documentation of all of the following is required:
  • appropriate diagnosis; and
  • results from genetic testing showing mutations in the beta glucuronidase gene; and
  • member's current weight.

miglustat

• Documentation of all of the following is required:
  • appropriate diagnosis; and
  • member is ≥ 18 years of age; and
  • results from enzyme assay test showing reduced activity of glucocerebrosidase; and
  • contraindication to enzyme replacement therapy.

Naglazyme

• Documentation of all of the following is required:
  • appropriate diagnosis; and
  • results from an enzyme assay test showing reduced arylsulfatase B (ASB) enzyme activity in leukocytes or fibroblasts along with elevated urine glycosaminoglycan (GAG) levels; and
  • member's current weight.

Palynziq

• Documentation of all of the following is required:
  • appropriate diagnosis; and
  • member is ≥ 18 years of age; and
  • results from genetic testing or molecular analysis to confirm diagnosis; and
  • blood phenylalanine concentrations > 600 micromol/L; and
  • medication will be used in conjunction with a phenylalanine-restricted diet; and
  • inadequate response, adverse reaction, or contraindication to sapropterin.

Ravicti

• Documentation of all of the following is required:
  • appropriate diagnosis; and
  • results from genetic test or an enzyme assay test supporting the diagnosis; and
  • member is ≥ two years of age; and
  • appropriate dosing; and
  • inadequate response, adverse reaction, or contraindication to sodium phenylbutyrate.