Skip to Content

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

Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

Drug
Notes

agalsidase beta Fabrazyme PA  
alglucosidase alfa Lumizyme PA   MB
asfotase alfa Strensiq PA  
avalglucosidase alfa-ngpt Nexviazyme PA   MB
elapegademase-lvlr Revcovi PA  
elosulfase alfa Vimizim PA   MB
galsulfase Naglazyme PA   MB
idursulfase Elaprase PA   MB
imiglucerase Cerezyme PA   MB
laronidase Aldurazyme PA   MB
olipudase alfa-rpcp Xenpozyme PA   MB
pegunigalsidase alfa-iwxj Elfabrio PA  
pegvaliase-pqpz Palynziq PA  
plasminogen, human-tvmh Ryplazim PA  
taliglucerase alfa Elelyso PA   MB
velaglucerase alfa Vpriv PA   MB
velmanase alfa-tycv Lamzede PA   MB
vestronidase alfa-vjbk Mepsevii PA   MB

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.

  • Lysosomal storage disorders are caused by a deficiency or absence of required enzymes. The consequence is an accumulation of compounds that are normally degraded, causing cell and organ dysfunction. Before the development of enzyme replacement therapy, management of these conditions consisted of supportive care and treatment of the complications.
  • A number of exogenously supplied enzymes are available for lysosomal storage disorders, including adenosine deaminase (ADA) deficiency, Gaucher disease, Fabry disease, Hunter syndrome, hyperammonemia due to the deficiency of the hepatic enzyme N-acetylglutamate synthetase, hypophosphatasia, lysosomal acid lipase deficiency, mucopolysaccharidosis type I, IVA, VI, and VII, non-central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) type B or A/B and Pompe disease.
  • Pancreatic enzyme replacement is indicated for the treatment of exocrine pancreatic insufficiency due to cystic fibrosis or other conditions. Multiple formulations of pancreatic enzymes exist with different combinations of lipase, protease, and amylase; however, these enzymes may differ in their effects. Members should be reevaluated after any changes in enzyme preparation or dose.
  • Molybdenum cofactor deficiency (MoCD) is a rare genetic disorder that results from one of several single gene defects in the biosynthetic pathway of molybdenum cofactor. About two-thirds of members have MoCD type A, which involves mutations in molybdenum cofactor synthesis gene 1 (MOSC1). Prior to the approval of fosdenopterin, the only available treatment options included supportive care and therapies directed towards complications arising from the disease. 
  • Pyruvate kinase deficiency is an inherited red blood cell enzyme disorder that causes chronic hemolysis. Affected individuals are either homozygous for a single pathogenic mutation or compound heterozygous for two different pathogenic variants affecting the function of the pyruvate kinase enzyme in red blood cells and liver. Mitapivat is a pyruvate kinase activator that acts by allosterically binding to the pyruvate kinase tetramer and increasing pyruvate kinase activity.
  • PIK3CA-Related Overgrowth Spectrum (PROS) is considered a rare disease that includes a group of genetic disorders, which leads to overgrowth of various body parts due to PIK3CA mutations. Alpelisib is small-molecule inhibitor of phosphatidylinositol-3 kinase (PI3K). Mutations in the gene for PI3K lead to PI3Ka and Akt activation, tumor generation, and cellular transformation. Activating these mutations lead to a range of malformations and overgrowths known as PROS. Alpelisib can inhibit the phosphorylation of PI3K and Akt to prevent further activity in the pathway.
  • ASMD is a rare autosomal recessive liposomal storage disease that results in a deficiency in the enzyme acid sphingomyelinase (ASM), which is required to metabolize sphingomyelin, a fatty acid. As a result, sphingomyelin accumulates in cells within major organs. Prior to the approval of olipudase alpha-rpcp, the only available treatment options included supportive care and therapies directed towards complications arising from the disease. Olipudase alfa-rpcp is the first and only FDA-approved drug to treat the underlying pathology of ASMD. This drug does not cross the blood-brain barrier; therefore, it is not expected to modulate the CNS manifestations of ASMD. 
  • Plasminogen deficiency (PLGD) type 1, also referred to as hypoplasminogenemia, is an ultra-rare, autosomal recessive disorder that can impair normal tissue and organ function which can lead to blindness. Individuals with PLGD type 1 lack the enzyme plasminogen and develop thick lesions in the mucous membranes of their body. There have been no standardized treatments for patients with PLGD due to the rarity of the disease, and plasminogen human-tvhm is the first and only product FDA-approved for the treatment of PLGD type 1. 
 

 Enzyme and Metabolic Disorder Therapies – Oral Agents

Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

Drug
Notes

alpelisib-Vijoice Vijoice PA  
carglumic acid Carbaglu PD PA   BP, A90
glycerol phenylbutyrate Ravicti PA  
leniolisib Joenja PA  
migalastat Galafold PA  
mitapivat Pyrukynd PA  
pancrelipase-Creon DR Creon DR test  
pancrelipase-Pertzye DR Pertzye DR test  
pancrelipase-Viokace Viokace test  
pancrelipase-Zenpep DR Zenpep DR test  
penicillamine capsule Cuprimine test   BP, A90
penicillamine tablet Depen test   # , A90
sacrosidase Sucraid PA  
sapropterin Kuvan PA  
sodium phenylbutyrate granules Pheburane PA  
sodium phenylbutyrate pellets for suspension Olpruva PA  
sodium phenylbutyrate powder, tablet Buphenyl test   BP, A90
trientine capsule Syprine test   BP, A90
trientine tablet Cuvrior PA  
triheptanoin Dojolvi PA  
uridine triacetate Xuriden PA  

 Enzyme and Metabolic Disorder Therapies – Substrate Replacement/Reduction Therapies

Drug Details

Drug Generic Name

Drug Brand Name

PA
Status

Drug
Notes

eliglustat Cerdelga PA  
fosdenopterin Nulibry PA   MB
miglustat Zavesca PA   BP
sebelipase alfa Kanuma PA   MB
Table Footnotes
# 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.

Back to top


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
    • 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 diagnosisand
    • 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.

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
    • 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


Clinical Criteria Main Page | Back to topPrevious  |  Next

Last updated 03/04/24

Feedback