Table 65: Enzyme and Metabolic Disorder Therapies
A B C D E F G H I J K L M N O P Q R S T U V W X Y Z
Drug Category: Endocrine and Metabolic Agents
Medication Class/Individual Agents: Enzyme and Metabolic Disorder Therapies
I. Prior-Authorization Requirements
Enzyme and Metabolic Disorder Therapies – Injectable Agents |
Clinical Notes |
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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.
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Enzyme and Metabolic Disorder Therapies – Oral Agents |
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Enzyme and Metabolic Disorder Therapies – Substrate Replacement/Reduction Therapies |
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| # | This designates a brand-name drug with FDA “A”-rated generic equivalents. PA is required for the brand, unless a particular form of that drug (for example, tablet, capsule, or liquid) does not have an FDA “A”-rated generic equivalent. |
| BP | Brand Preferred over generic equivalents. In general, MassHealth requires a trial of the preferred drug or clinical rationale for prescribing the non-preferred drug generic equivalent. |
| PD | Preferred Drug. In general, MassHealth requires a trial of the preferred drug or clinical rationale for prescribing a non-preferred drug within a therapeutic class. |
| MB | This drug is available through the health care professional who administers the drug or in an outpatient or inpatient hospital setting. MassHealth does not pay for this drug to be dispensed through the retail pharmacy. If listed, PA does not apply through the hospital outpatient and inpatient settings. Please refer to 130 CMR 433.408 for PA requirements for other health care professionals. Notwithstanding the above, this drug may be an exception to the unified pharmacy policy; please refer to respective MassHealth Accountable Care Partnership Plans (ACPPs) and Managed Care Organizations (MCOs) for PA status and criteria, if applicable. |
| A90 | Allowable 90-day supply. Dispensing in up to a 90-day supply is allowed. May not include all strengths or formulations. Quantity limits and other restrictions may apply. |
II. Therapeutic Uses
FDA-approved, for example:
- Acid sphingomyelinase deficiency (ASMD) (Xenpozyme)
- Activated phosphoinositide 3-kinase delta (PI3Kd) syndrome (APDS) (Joenja)
- Adenosine deaminase severe combined immunodeficiency (ADA-SCID) (Revcovi)
- Alpha-mannosidosis (Lamzede)
- Congenital sucrase-isomaltase deficiency (Sucraid)
- Fabry disease (Elfabrio, Fabrazyme, Galafold)
- Gaucher Disease Type 1 (Cerdelga, Cerezyme, Elelyso, miglustat, Vpriv)
- Hemolytic anemia with pyruvate kinase deficiency (Pyrukynd)
- Hereditary orotic aciduria (Xuriden)
- Hunter Syndrome (Elaprase)
- Hyperammonemia due to the deficiency of the hepatic enzyme N-acetylglutamate synthetase (NAGS) (carglumic acid)
- Hyperammonemia due to propionic aciduria (PA) or methylmalonic aciduria (MMA) (carglumic acid)
- Hypophosphatasia (Strensiq)
- Long-chain fatty acid oxidation disorders (LC-FAOD) (Dojolvi)
- Lysosomal acid lipase deficiency (Kanuma)
- Molybdenum cofactor deficiency (MoCD) Type A (Nulibry)
- Mucopolysaccharidosis I (Aldurazyme)
- Mucopolysaccharidosis IVA (Morquio A syndrome) (Vimizim)
- Mucopolysaccharidosis VI (Naglazyme)
- Mucopolysaccharidosis VII (Sly syndrome) (Mepsevii)
- Phenylketonuria (Palynziq, sapropterin)
- PIK3CA-Related Overgrowth Spectrum (PROS) (Vijoice)
- Plasminogen deficiency (PLGD), Type 1 (Ryplazim)
- Pompe disease (Lumizyme, Nexviazyme)
- Urea cycle disorder (Olpruva, Pheburane, Ravicti)
- Wilson’s disease (Cuvrior)
Non-FDA approved, for example:
- Acute hyperammonemia in isovaleric aciduria (carglumic acid)
Note: The above lists may not include all FDA-approved and non-FDA approved indications.
III. Evaluation Criteria for Approval
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.
- Dispensing in a 90-day supply of medication may be mandated or allowable for agents in this therapeutic class (designated by M90 or A90, respectively, in the Drug Notes section above). Applicable quantity limits are described below as units per day, per month, per 28 days, or as clinically appropriate, and may be extrapolated for fills of longer day supply.
- 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.
- For recertification requests, approval may require submission of additional documentation including, but not limited to, documentation of: some or all criteria for the original approval; response to therapy; clinical rationale for continuation of use; status of member’s condition; appropriate diagnosis; appropriate age; appropriate dose, frequency, and duration of use for requested medication; complete treatment plan; current laboratory values; and member’s current weight.
- Additional criteria may apply depending upon diagnosis and/or requested medication (see below). Other factors, including rebate and FDA-approval status, may change independently of scheduled MassHealth updates, which may result in additional restrictions.
Aldurazyme
- Documentation of all of the following is required:
- appropriate diagnosis; and
- results from genetic testing showing mutations in IDUA gene or an enzyme assay test showing reduced lysosomal alpha-L-iduronidase activity in peripheral blood leukocytes, plasma, or cultured fibroblasts; and
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; and
- member's current weight.
carglumic acid
- Documentation of all of the following is required for hyperammonemia due to NAGS deficiency:
- appropriate diagnosis; and
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; and
- appropriate dosing; and
- results from genetic test or an enzyme assay test supporting the diagnosis.
- Documentation of all of the following is required for hyperammonemia due to PA or MMA:
- appropriate diagnosis; and
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; and
- appropriate dosing; and
- results from genetic testing, medical records, or lab results supporting the diagnosis; and
- elevated ammonia levels > 60 μmol/L.
- Documentation of all of the following is required for acute hyperammonemia in isovaleric aciduria (IVA):
- appropriate diagnosis; and
- medical records and/or laboratory testing results supporting the diagnosis of IVA; and
- abnormally elevated baseline ammonia levels (e.g., > 60 μmol/L); and
- appropriate dosing.
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
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; 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 genetic test confirming mutation in GBA gene or an enzyme assay test showing reduced activity of the enzyme glucocerebrosidase; and
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; and
- member's current weight.
Cuvrior
- Documentation of all of the following is required:
- appropriate diagnosis; and
- member is ≥18 years of age; and
- member has stable disease; and
- member is tolerant to penicillamine; and
- contraindication to penicillamine; and
- inadequate response, adverse reaction, or contraindication to trientine capsule; and
- requested medication will not be taken concurrently with penicillamine; and
- appropriate dosing.
Dojolvi
- Documentation of all of the following is required:
- diagnosis of long-chain fatty acid oxidation disorders (LC-FAOD); and
- results from genetic testing or molecular analysis to confirm diagnosis (e.g., CPT I or II, LCHAD, TFP, VLCAD deficiency); and
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; and
- trial with a diet consisting of low-fat, high-carbohydrates, and avoidance of fasting; and
- member's current caloric intake.
Elaprase
- Documentation of all of the following is required:
- appropriate diagnosis; and
- results from genetic testing confirming mutation in IDS gene or iduronate-2-sulfatase assay test showing reduced or absent activity in the serum, white blood cells, or fibroblasts; and
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; and
- member's current weight.
Elelyso
- Documentation of all of the following is required:
- appropriate diagnosis; and
- results from genetic test confirming mutation in GBA gene or an enzyme assay test showing reduced activity of the enzyme glucocerebrosidase; and
- member is ≥ four years of age; and
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; and
- member's current weight.
Elfabrio and Fabrazyme
- Documentation of all of the following is required:
- appropriate diagnosis; and
- one of the following:
- results from an enzyme assay test showing reduced or absent α-galactosidase A (α-GAL) enzyme activity in plasma, leukocytes, tears, or biopsied tissue; or
- Genetic testing confirming mutation in GAL gene; or
- Biomarker demonstrating an increase in Gb3 concentration; and
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; and
- member's current weight; and
- for Elfabrio, inadequate response, adverse reaction, or contraindication to Fabrazyme.
Joenja
- Documentation of all of the following is required:
- appropriate diagnosis; and
- member is ≥ 12 years of age; and
- prescriber is a specialist (e.g., pediatrician, hematologist/oncologist, or allergist/immunologist), or consult notes from a specialist are provided; and
- results from genetic testing confirming mutation in the PIK3CD or PIK3R1 genes; and
- member's weight is ≥ 45 kg; and
- appropriate dosing.
Galafold
- Documentation of all of the following is required:
- appropriate diagnosis; and
- member is ≥ 18 years of age; and
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are 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
- requested quantity is ≤ 15 units/30 days.
Kanuma
- Documentation of all of the following is required:
- diagnosis of lysosomal acid lipase deficiency; and
- one of the following:
- lab assay documenting low lysosomal acid lipase activity; or
- genetic testing confirming full or partial loss of LAL gene; and
- prescriber is a specialist in genetic or metabolic diseases or consult notes from a specialist are provided; and
- member's current weight.
Lamzede
- Documentation of all of the following is required:
- appropriate diagnosis; and
- member is ≥ 3 years of age; and
- copy of a genetic test confirming diagnosis of alpha-mannosidosis (e.g., mutation of MAN2B1 gene); and
- baseline measurements for all of the following tests:
- one of the following motor function tests:
- 3-minute stair climb test; or
- 6-minute walk test; and
- serum oligosaccharides; and
- forced vital capacity; and
- one of the following motor function tests:
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; and
- member's current weight.
Lumizyme
- Documentation of all of the following is required:
- appropriate diagnosis; and
- one of the following:
- results from acid maltase enzyme alpha-glucosidase (GAA) assay test showing reduced or absent activity from cultured skin fibroblasts; or
- lymphocyte testing; or
- blood spot assay; or
- genetic testing confirming mutation in GAA gene; and
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; 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
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; 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
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; 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
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; and
- member's current weight.
Nexviazyme
- Documentation of all of the following is required:
- appropriate diagnosis; and
- one of the following:
- results from GAA assay test showing reduced or absent activity from cultured skin fibroblasts; or
- lymphocyte testing; or
- blood spot assay; or
- genetic testing confirming mutation in GAA gene; and
- member is ≥ one year of age; and
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; and
- member's current weight; and
- for members weighing < 30 kg, contraindication to Lumizyme.
Nulibry
- Documentation of all of the following is required:
- appropriate diagnosis confirmed by genetic testing; and
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; and
- appropriate dosing; and
- member's current weight.
Olpruva
- Documentation of all of the following is required:
- appropriate diagnosis; and
- results from genetic test or an enzyme assay test supporting the diagnosis; and
- prescriber is a specialist in genetic or metabolic diseases or consult notes from a specialist are provided; and
- appropriate dosing; and
- inadequate response or adverse reaction to one or contraindication to both of the following: sodium phenylbutyrate powder, sodium phenylbutyrate tablet; and
- inadequate response, adverse reaction, or contraindication to Pheburane.
Palynziq
- Documentation of all of the following is required:
- appropriate diagnosis; and
- member is ≥ 18 years of age; and
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; 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.
Pheburane
- Documentation of all of the following is required:
- appropriate diagnosis; and
- results from genetic test or an enzyme assay test supporting the diagnosis; and
- prescriber is a specialist in genetic or metabolic diseases or consult notes from a specialist are provided; and
- appropriate dosing; and
- inadequate response or adverse reaction to one or contraindication to both of the following: sodium phenylbutyrate powder, sodium phenylbutyrate tablet.
Pyrukynd
- Documentation of all of the following is required:
- diagnosis of hemolytic anemia with pyruvate kinase deficiency; and
- member is ≥ 18 years of age; and
- results from genetic testing confirming mutation in PKLR gene or lab testing showing reduced or absent activity of pyruvate kinase; and
- prescriber is a specialist in genetic diseases, hematology, or metabolic diseases or consultation notes from a specialist are provided; and
- hemoglobin (Hb) ≤ 10 g/dL (dated within the last 60 days); and
- requested quantity is ≤ two units/day.
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
- prescriber is a specialist in genetic or metabolic diseases or consult notes from a specialist are provided; and
- appropriate dosing; and
- inadequate response or adverse reaction to one or contraindication to all of the following: sodium phenylbutyrate powder, sodium phenylbutyrate tablet, Olpruva, Pheburane.
Revcovi
- 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
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; and
- member's current weight.
Ryplazim
- Documentation of all of the following is required:
- appropriate diagnosis; and
- history of lesions (external and/or internal) and symptoms consistent with a diagnosis of PLGD type 1; and
- baseline plasminogen activity level ≤ 45%; and
- one of the following:
- results from genetic testing showing mutations in PLG gene; or
- member has plasminogen antigen levels ≤ 9 mg/dL; and
- requested dose is ≤ 6.6 mg/kg every two to four days.
sapropterin
- Documentation of all of the following is required:
- appropriate diagnosis; and
- documentation that medication will be used in conjunction with a phenylalanine-restricted diet; and
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; and
- member's current weight.
Strensiq
- Documentation of all of the following is required:
- diagnosis of perinatal-onset, infantile-onset, or juvenile-onset hypophosphatasia; and
- genetic testing confirming mutation in ALPL gene; and
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; and
- member's current weight.
Sucraid
- Documentation of all of the following is required:
- diagnosis of congenital sucrase-isomaltase deficiency (CSID); and
- results from small bowel biopsy or breath hydrogen test showing reduced or absent enzyme activity or sucrase breath test; and
- prescriber is a specialist in genetic or metabolic diseases, a gastroenterologist, or consult notes from a specialist or gastroenterologist are provided; and
- member's current weight.
Vijoice
- Documentation of all of the following is required:
- diagnosis of PROS with congenital or early childhood onset; and
- member is ≥ two years of age; and
- overgrowth is sporadic and mosaic (i.e., patchy, irregular); and
- results from genetic testing showing evidence of a mutation in the PIK3CA gene; and
- appropriate dosing; and
- medical records documenting one of the following:
- spectrum categorization defined as having at least two of the following:
- adipose, muscle, nerve, or skeletal overgrowth; or
- capillary, venous, arteriovenous, or lymphatic vascular malformations; or
- epidermal nevus; or
- isolated features defined as having one of the following:
- large isolated lymphatic malformation; or
- isolated macrodactyly or overgrown splayed feet/hands, overgrown limbs; or
- truncal adipose overgrowth; or
- bilateral hemimegalencephaly/dysplastic megalencephaly/focal cortical dysplasia type 2; or
- epidermal nevus; or
- seborrheic keratoses; or
- benign lichenoid keratoses.
- spectrum categorization defined as having at least two of the following:
Vimizim
- Documentation of all of the following is required:
- appropriate diagnosis; and
- member is ≥ five years of age; and
- results from an enzyme assay test showing reduced N-acetylgalactosamine-6-sulfatase activity in blood and/or skin cells; and
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; and
- member's current weight.
Xenpozyme
- Documentation of all of the following is required:
- diagnosis of non-central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) type B or ASMD type A/B; and
- prescriber is a specialist (e.g., medical geneticist or a specialist familiar with lysosomal storage disorders) or consult notes from a specialist are provided; and
- one of the following:
- for members ≥ 18 years of age, both of the following:
- DLco ≤ 70% of predicted normal value; and
- spleen volume ≥ 6 MN; or
- for members < 18 years of age, spleen volume ≥ 5 MN; and
- for members ≥ 18 years of age, both of the following:
- member does not have acute or rapidly progressing neurologic abnormalities; and
- both of the following:
- member does not require invasive ventilatory support; and
- member does not require noninvasive ventilatory support while awake for > 12 hours a day; and
- member's current weight; and
- appropriate dosing.
- For recertification, documentation of all of the following is required:
- improvement from baseline in DLco and spleen volume; and
- updated member weight.
Xuriden
- Documentation of all of the following is required:
- diagnosis of hereditary orotic aciduria (HOA); and
- genetic testing confirming mutation in UMPS gene; and
- prescriber is a specialist in genetic or metabolic diseases or consultation notes from a specialist are provided; and
- member's current weight.
Original Effective Date: 11/2012
Last Revised Date: 03/2024
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Last updated 04/01/24